An Invitation to participate in “MG and Work” survey

The MAA has partnered with the University of the Sunshine Coast to conduct research on Myasthenia Gravis and people’s ability to work. The research team is led by Dr. Michele Verdonck.

MG can significantly affect people’s ability to work. Little is understood about the factors that support or hinder people with Myasthenia wanting to work. We are seeking your valued input as a person living with MG. We are conducting research aimed at investigating the factors which impact someone who is living with MG and their ability to engage in and maintain paid work.

Participants are invited to complete an anonymous online survey, which will take approximately 30 minutes to complete. We are hoping to use the findings from this research to help improve the working lives of those diagnosed with MG through identifying the enablers and barriers commonly experienced.

For more detailed information and to access the survey, please use this link: https://uniofsunshinecoast.syd1.qualtrics.com/jfe/form/SV_b8zGLyrsXJWYEwC

Please also find attached the Research Project Information Sheet for more details. If you have any questions or concerns, please contact the Chief Investigator, Michele Verdonck, using michele.verdonck@usc.edu.au. If someone you know may also be interesting in participating in this study, please feel free to share this email.

Ethics Approval: S242004

Click here to download the Research Project Information Sheet

10 Years celebrating Advocacy, Research, and Unity

The MAA is proudly celebrating ten years with all our keen supporters by sharing a few words from the three founding members who have been an integral part of this wonderful decade of the alliance.

Please do view the timeline included here. It reflects the key components of our history. We are sure that you will find the variety of content inspiring and diverse.

Thank you to all our MG Community for their continued support.

Together we are a strong and influencing voice!

“Who would have thought ……..” is my catchcry! It has been an amazing ten years of new, interesting and purposeful experiences where we are confident that we have made a difference for the Australian Myasthenia community.

It has also been a joy to work with such wonderful team members. A small but mighty group. Each volunteer offers so much to this organisation and they offer so much of themselves and their time.

There is great potential for the future endeavours of the MAA.

Susan

After a very detailed and concise planning process the Myasthenia Alliance Australia (MAA) was founded in 2014.  This group, which had a clear focus on the national agenda, was made up of the incorporated state associations.

As a foundation member of the MAA I have seen it grow from very humble beginnings to a highly strategic group with strong affiliations with government, pharmaceutical companies and other not-for-profit advocacy organizations.

The work of this alliance cannot be underestimated.  The work with the state associations to raise needed funds for the ‘build’ of MGBase, the ongoing advocacy with members of parliament in relation to the provision of services and products for people living with MG, the ever-increasing  recognition by pharmaceutical companies, the strong affiliation with both internationally and nationally recognised lead neurologists and the involvement in a wide breadth of research are just a broad indication of what the MAA has, and continues to, achieve.

I am very proud to have been a part of such a widely-recognised and proactive small group of individuals constantly working to improve the lives of people living with MG and their families.  I know that the MAA will gain further recognition into the future.

Carol

As a founding member, starting out I did not know what to expect, but very pleased we are still going strong and are supporting the MG community.

Glenda

 

CLOSING SOON! Market research on Generalized Myasthenia Gravis (gMG)

Help Shape the Future of gMG Care – Market Research Closing Soon!

The MAA respectfully requests patient support in further telling the story of their experiences with all forms of Myasthenia. Please note, we are very pleased that this survey can be completed via a hardcopy option. Please call Libby on 0407 110 204 to arrange this.

Late last year Alexion asked several people living with MG to participate in an online insight gathering project that aimed to better understand the current patient and doctor dynamics so that they can help support people living with gMG by addressing unmet needs at different points in time along the patient journey.

Several members of our community put up their hands and shared their experience which was incredibly important in shaping a real world understanding of life with MG and has assisted Alexion’s efforts to bring access to new treatment options.

The main takeaways identified gaps in what patients may need or expect and their clinical experience:

  • The disconnect between HCPs and people living with MG is very real
  • All people living with MG interviewed recall difficulties and challenges with HCPs at some point in their journey – especially early on
  • Some doctors can downplay the impact of MG which may cause them to appear ignorant or dismissive
  • People living with MG can feel underwhelmed with their healthcare experiences, under-prepared and forced to advocate for themselves
  • People living with MG who are not managed by MG specialists can face a more challenging journey
  • The physical and emotional rollercoaster of MG can leave many people living with MG feeling isolated and socially outcast
  • Managing side effects can prevent people living with MG from reaching their own personal goal of wanting to just feel “normal”
  • It can take years to get to a point where people living with MG feel some level of satisfaction with treatment
  • Doctors are focused on clinical markers to judge success more often than what a person living with MG might consider their own treatment or health goal

Whilst much of what was shared and learned from the exercise will not be new to many of you, the findings have helped Alexion to develop a more robust survey that will help more people living with MG validate the gaps raised in the initial phase of the project.  

The purpose of this survey is to get to an even deeper understanding of the impact of gMG on the lives people living with MG and validate the gaps needs.

The self-completed online survey will take participants about 15 minutes. We have partnered with Elbow Insights and Strategy, a market research agency that specialises in this area, to help us develop and distribute the survey.

We are hoping we can have your help in sharing this research with community members so that those interested can participate in the survey.

If you are interested in helping us, you can use the link below to preview the survey or contact us to get your personalised link. Please note participants who use the link below needs to be completed in one sitting.

https://rebrand.ly/1816Pearl2

If you are unable to do the survey online, then please reach out to Libby @ Alexion on libby.noble@alexion.com or text her on 0407 110 204, and she will organize to send you a hard copy.

Market research on Generalized Myasthenia Gravis (gMG)

Dear MGNSW community

The MAA (Myasthenia Alliance Australia) respectfully requests patient support in further telling the story of their experiences with all forms of Myasthenia. Please note, we are very pleased that this survey can be completed via a hardcopy option. Please call Libby on 0407 110 204 to arrange this.

Late last year Alexion asked several people living with MG to participate in an online insight gathering project that aimed to better understand the current patient and doctor dynamics so that they can help support people living with gMG by addressing unmet needs at different points in time along the patient journey.

Several members of our community put up their hands and shared their experience, which was incredibly important in shaping a real world understanding of life with MG and has assisted Alexion’s efforts to bring access to new treatment options.

The main takeaways identified gaps in what patients may need or expect and their clinical experience:

  • The disconnect between HCPs and people living with MG is very real
  • All people living with MG interviewed recall difficulties and challenges with HCPs at some point in their journey – especially early on
  • Some doctors can downplay the impact of MG which may cause them to appear ignorant or dismissive
  • People living with MG can feel underwhelmed with their healthcare experiences, under-prepared and forced to advocate for themselves
  • People living with MG who are not managed by MG specialists can face a more challenging journey
  • The physical and emotional rollercoaster of MG can leave many people living with MG feeling isolated and socially outcast
  • Managing side effects can prevent people living with MG from reaching their own personal goal of wanting to just feel “normal”
  • It can take years to get to a point where people living with MG feel some level of satisfaction with treatment
  • Doctors are focused on clinical markers to judge success more often than what a person living with MG might consider their own treatment or health goal

Whilst much of what was shared and learned from the exercise will not be new to many of you, the findings have helped Alexion to develop a more robust survey that will help more people living with MG validate the gaps raised in the initial phase of the project.

The purpose of this survey is to get to an even deeper understanding of the impact of gMG on the lives people living with MG and validate the gaps needs.

The self-completed online survey will take participants about 15 minutes. We have partnered with Elbow Insights and Strategy, a market research agency that specialises in this area, to help us develop and distribute the survey.

We are hoping we can have your help in sharing this research with community members so that those interested can participate in the survey.

If you are interested in helping us, you can use the link below to preview the survey or contact us to get your personalised link. Please note participants who use the link below needs to be completed in one sitting.

https://rebrand.ly/1816Pearl2

If you are unable to do the survey online, then please reach out to Libby @ Alexion on libby.noble@alexion.com or text her on 0407 110 204, and she will organize to send you a hard copy.

 

PBAC Decision: New MG Medications Not Recommended, But Advocacy Efforts Continue

Recently the Pharmaceutical Benefits Advisory Committee (PBAC) announced the outcome of their July Meeting. The MG community were keenly waiting to hear the PBAC Outcomes as two new medications, Raviluzimab and Zilucoplan, had been reviewed. Regrettably both medications received a ”Not Recommended” verdict. The PBAC did recognise the high clinical need for effective therapies for gMG.

Whilst extremely disappointing it is not the end of the process. Alexion assures the MAA that they are committed to working with the PBAC alongside the MG Clinical and patient communities to bring access at the earliest opportunity to their treatment.

Both Pharmaceutical companies (Alexion and UCB) will resubmit in 2025.

Please read below for a statement from Libby Noble at Alexion.

Patient Advocacy Director, Libby Noble told us: “We had hoped for a different outcome and we understand it’s both upsetting and frustrating to many who struggle with this condition and especially to those who took the time to share their views to the PBAC. We want to extend our sincere gratitude to MAA and the MG community for their invaluable input and support throughout this process. On a positive note, we are pleased that the PBAC recognised the high clinical need for effective therapies for MG, particularly in patients who are not responding to or are unable to use existing therapies, and for those with refractory disease. That recognition is testament to the lived experience shared by patients and caregivers that helped to shape that understanding, and sets a strong foundation for our continued efforts to bring access to much needed new treatment options”.

Alexion are working on their next steps and will share an update on timings with us as soon as possible. They would also like to invite the community to join a webinar on Friday 11 October to share the PBAC outcomes in greater detail, address community concerns, and update on their continued efforts to advocate for timely and equitable access in Mg.

More information will be shared regarding the webinar as it comes to hand.

Gene Therapy for Congenital Myasthenia Syndrome – Webinar Invitation

 

The MAA first engaged with Amplo Biotechnologies at the 2019 Brisbane Conference. We are delighted to report that Patricio Sepulveda, CEO, is offering to share updated information on “what they are doing and where they are at”. Patricio reports that 35 different genes have now been identified to capture more people with Congenital Myasthenia Syndrome. This knowledge is important in providing the correct treatment. Patients, Clinicians and Carers are invited to attend the following information sessions. Australians can join at either 7am on August 14th (choose the US option) or 7pm on August 14th (choose the UK option). The session is expected to run for 45 minutes. Written questions will be answered on the day or by follow-up email.

Register utilising the QR code or link found in the flyer below. If you are sero-negative MG, please consider joining the webinar as some patients are being diagnosed quite late in life.

AMPLO IS DELIGHTED TO SHARE THE LATEST ADVANCEMENTS IN ITS GENE THERAPY FOR CONGENITAL MYASTHENIC SYNDROME. THIS IS A FREE WEBINAR FOR PATIENTS, CARERS AND CLINICIANS

Follow the link to register:

Tuesday 13th Aug, 5pm US EST
Wednesday 14th Aug, 10 am UK/GMT+1

 

Partnering With Canada

To Australians with Myasthenia, the following is a message from Dr Carolina Barnette-Tapia MD, PhD of Canada.

The MAA thank Australians for their interest in supporting this project. Responses will be welcomed until early September 2024. Information about the outcomes resulting from this research has been requested.

Disclosure information is provided after logging in.

“We invite you to participate in this international study to assess how people with Myasthenia living in different countries make treatment decisions and which aspects of treatment are more important to them. We kindly thank the MAA for supporting our study, which will help understand if the preferences of Australians living with MG are different to those from other countries.

Carolina Barnett-Tapia, MD, PhD
Associate Professor of Medicine (Neurology), University of Toronto
Ellen and Martin Prosserman Centre for Neuromuscular Diseases
Elisabeth Raab Neurofibromatosis Clinic
Toronto General Hospital / UHN”

To participate, scan the QR code below or click here.

 

RESEARCH REPORT!

The Myasthenia Alliance Australia (MAA) supports and promotes research that is specific to the experience of Myasthenia Gravis (MG) patients in Australia. But we would not be able to do this without the essential and valuable contribution of Australians with MG.

One current project is VALUE-Ig, which aims to generate evidence to inform the optimal use of immunoglobulin (Ig) in four patient cohorts, including MG. This study is being conducted by a research team at Monash University Centre for Health Economics and is funded by the Australian Government’s Medical Research Future Fund.

VALUE-Ig includes a Discrete Choice Experiment (DCE) to explore the treatment preferences of MG patients in Australia. The first round of focus groups to help design this DCE study has been completed and we sincerely thank the MG patients who gave their time and insights to this important research. Please read below for an update from the VALUE-Ig research team. To find out more, you can contact the team directly at value.ig@monash.edu or reach out to the MAA by calling 1800 802 568 or emailing info@mgaq.org.au or info@myasthenia.org.au.

The researchers thank all the MG patients who participated in the first focus group sessions. Sharing their lived experiences helped us better understand MG and design our patient preferences survey, which will be launched in 2025. Overall, 19 patients joined the sessions, ranging in age from 35 to 82. Among them, 12 were female and 7 were male. The patients were from various regions: 9 from NSW, 5 from Vic, 4 from QLD, and 1 from WA.

They frequently highlighted the importance of improving healthcare workers’ knowledge of MG and the availability of neurologists to ensure timely access to necessary treatments. There were also discussions about how location impacted the accessibility of health services. Especially those living in regional areas felt that they had less options for accessing treatments.

They helped us revise the descriptions of our hypothetical scenarios that will be presented to respondents of our future survey. During the sessions, MG patients discussed their priorities when receiving treatment for their MG in mild exacerbation, severe exacerbation, and maintenance treatment conditions. They listed the most commonly experienced treatment side effects and identified which side effects were most important to them when receiving treatment. Gastrointestinal side effects were the most consistently reported, while the side effects patients were most concerned about varied widely. Reported side effects of MG medications included fatigue, weight gain, depression, line infection risk, blood clotting risk, increased risk of skin cancer and diabetes, hair loss, bone loss and poor sleep. They acknowledged their ongoing need to balance the side effects of treatment and their MG symptoms.

Although it was not within the scope of our focus group sessions, it is important to note that the session evaluation survey results indicate that many MG patients are interested in learning more about new treatments available.

 

Monash University
Centre for Health Economics | Monash Business School
Level 5, Building H, Caulfield Campus
900 Dandenong Road
Caulfield East VIC 3145
Australia

Thank you to the Myasthenia Community

 

 

We  extend heartfelt gratitude to every member of our community, as well as to the family and friends whose unwavering support made the recent MAA’s Art With Heart project a resounding success.

The second installment of the Art With Heart Awareness Campaign has concluded, and the MAA Board is thrilled with the outcome. The auction featured a delightful array of items generously contributed by supporters. We extend our sincere thanks to Hermann Pekel and Chris Searle, esteemed Australian artists whose generous contributions enriched this campaign.

The response from donors was exceptionally gratifying, reflecting broad community engagement and support. This increased awareness was amplified through various channels, including radio coverage, expanding our reach significantly. The proceeds generated will further MAA’s advocacy efforts, fueling plans for even larger campaigns in the future.

Make a submission to PBAC about a new complement inhibitor treatment, Zilucoplan

A new treatment for AChR+ generalised MG, Zilucoplan (brand name Zilbrysq ®) is on the agenda for the next Pharmaceutical Benefits Advisory Committee (PBAC) meeting.

Make a personal submission highlighting the lived experience as a MG patient by completing the online form here by Wednesday 29 May 2024.

A submission is essentially about sharing your story of living with MG with national decision makers as they consider the benefits, costs, and delivery of new treatments. As we are quite a diverse bunch of people and each of our journeys is a bit different, an individual submission helps demonstrate the importance of having different treatment options to give us all the best care and quality of life.

Recent research and patient feedback show that there are still unmet needs and a burden for MG patients in Australia. The vision of the MAA is timely and equitable access to the best available treatment that most benefits each patient. We hope to achieve this by ensuring treatment options are available, accessible, and affordable.

Please read the attached guidance document for more information. If you have any questions, contact us at:

For more details, click the link below:

PBAC Consumer Submission for Zilbrysq (Zilucoplan) 2- May 2024

 

SEEKING FOCUS GROUP participants to help DESIGN RESEARCH for MG patients

.

The MAA is working with Monash University researchers on the VALUE-Ig project. We are looking for MG patients to participate in a focus group to help design a study about patient preferences on treatments. This is an opportunity to provide input before the research is carried out and we invite you to take part.

This research project aims to recruit patients diagnosed with Myasthenia Gravis for participation in focus group sessions. These focus groups aim to explore the preferences of people who have Myasthenia Gravis towards different treatment options.

You are invited to take part in 2 focus group sessions with up to 4 other volunteers, plus Monash researchers. The focus group will be an online conversation that will last approximately one hour, so please make sure you can access the internet to participate.

As a small token of appreciation for your time and insights, you may choose to receive a digital gift voucher ($50) for taking part in the 2 sessions.

Please see the attached brochure for more information.

If you are interested in participating in the study, have any questions or require more information, please contact the Monash University researchers directly via email: value.ig@monash.edu or phone: 03 9903 4363.

If you don’t want to miss this opportunity, be quick to contact the team as there are limited places to keep the focus groups small. Thank you for helping the MAA to build the research and evidence for MG in Australia!

 

 

VALUE-Ig Focus Group – PIF Online A4 brochure

ART WITH HEART Awareness Campaign is live!

 
The second ever ART WITH HEART Awareness Campaign is under way! The aim of this campaign is to share this opportunity with the general population and to familiarise them with the condition Myasthenia Gravis.
The MAA wants the general community to understand that whilst ‘fun-run events’ and ‘festivals in the sun’ don’t work for us, there is much that we do achieve and still love to participate in within our lives. This auction event is a time for our members to showcase their talents, to share with family and friends and to start conversations in new places. We hope that everyone will do their utmost to make the campaign a success.
What are we asking you to do to make this event effective? Firstly, please donate items or ask others if they would like to contribute. Donations are now being accepted and will be gathered until the end of May. 
Next, share the news as widely as possible. This can be done in conversation, via emails or sharing on Facebook, Instagram, etc. It is such an easy topic to share. We hope you will give it a try.
Finally, we need people to bid at the Auction when it opens in June.
Some wonderful items have already been donated to the Art With Heart campaign and you can see them by visiting the MAA website (and here’s a reminder to register for updates!): www.myastheniaalliance.org.au. We are so very grateful for the amazing support received from two renowned Australian artists who have gifted paintings to support people living with this rare disease. These artists are Herman Pekel and Chris Seale.
With knowledge and awareness, there is greater understanding of this rare condition and what it means to live with it. We hope for broader workplace options, better support in the general community, and more informed engagement with health care providers.
(Content shared on behalf of the MAA.)

UPDATE – THERE IS STILL TIME TO MAKE YOUR SUBMISSION TO PBAC

We hope the new year is off to a great start for you! The Myasthenia Alliance Australia (MAA) is sharing the attached information with you about the current opportunity to make a personal submission to the Pharmaceutical Benefits Advisory Committee (PBAC) about a new treatment called Ultomiris (Ravulizumab). 
 
Please read this in addition to the information we shared in December, which can also be accessed here: https://myastheniaalliance.org.au/information-about-making-a-pbac-submission-for-ravulizumab/
 
A reminder that the deadline for submissions is Wednesday, 31 January 2024. 
 
The Consumer Submission can be downloaded as a PDF from here:
The Consumer Submission can be downloaded as a Word document from here:
PBAC Consumer Submission for Ravulizumab – January 2024 (Part 2)

The Government’s Consumer Medicine Information (CMI) about Ultomiris (Ravulizumab) can be accessed via this link. 

Kind regards, 
The MAA Board

Myasthenia Gravis: New Drugs and a Road to Individualized Treatment – An article from Yale Medicine

An interesting article about the development of new myasthenia gravis treatments.  Please note that traditional treatments and their effectiveness are outside the scope of the article.

This article was originally published on www.yalemedicine.org  BY KATHY KATELLA NOVEMBER 17, 2023

Myasthenia gravis is a complex autoimmune disease that interferes with the communication between nerves and muscles. The hallmark symptom is muscle weakness that worsens after activity and improves with rest. It might start in the mouth or face muscles, causing slurring of speech or drooping eyelids. It also can affect other muscles that move the arms and legs, resulting in difficulty with walking or getting up from a chair.

For some people, a few of these symptoms are a minor annoyance; others, however, lose the ability to walk, talk, or even breathe properly. As a result, some people can’t attend school, care for a family, or hold a job.

Fortunately, there has been encouraging progress in treatments for the condition. “If we go even a few years back—prior to 2017—there were no Food and Drug Administration [FDA]-approved medications for myasthenia gravis,” says Yale Medicine neurologist Richard J. Nowak, MD, MS, founding director of the Yale Medicine Myasthenia Gravis Program. Treatments such as steroids, which had debilitating side effects, and other broad immunosuppressive therapies were prescribed off-label, meaning they were used for a purpose other than that for which they were initially approved.

Today, five FDA-approved medications for myasthenia gravis are helping more patients than before manage their symptoms and lead active lives. However, there are still some questions in many cases about which medicine will work best for which patients. “The science has moved forward so rapidly and deeply in understanding the disease that the new therapeutics can be applied with some level of confidence,” says Yale neuroimmunologist Kevin O’Connor, PhD, a National Institutes of Health (NIH)-funded researcher. At the same time, scientists have learned that different patients may, in fact, have pathologies due to different mechanisms, and a medicine that makes one patient symptom-free may not work at all for another, he adds.

Dr. Nowak treats patients with myasthenia gravis from Connecticut and beyond. The Yale Myasthenia Gravis Program, established over a decade ago, is one of the largest programs of its kind in the U.S. Dr. Nowak and O’Connor have been working together for over a dozen years to further research and provide better care for patients.

They answer questions (below) about how knowledge of myasthenia gravis is changing.

What is myasthenia gravis?

Myasthenia gravis is a rare, chronic neuromuscular disease that affects an estimated 75,000-100,000 people in the United States. “The disease is caused by autoantibodies that target proteins in the neuromuscular junction [where nerves and muscle fibers meet] so that the muscle does not receive the signal from the nerve, and, as a result, the muscle doesn’t move as it should,” O’Connor says. (While antibodies recognize foreign substances, such as viruses, and neutralize them, autoantibodies react to elements made by a person’s own body.)

This leads to muscular weakness and fatigue. The symptoms may affect only certain muscles, such as the eyelids, facial muscles, the muscles that affect speech, or the upper arms and legs. Myasthenia gravis can be ocular, appearing in the muscles that control eye movements, or generalized, affecting muscles around the eyes, mouth, arms, legs, and respiratory system.

The condition affects people of all ages, although it tends to become apparent in adulthood, and commonly impacts women under 40 and men over 60.

While most people with myasthenia gravis have moderate disease, about one-fifth have mild disease with a symptom such as eyelid drooping that is easily managed with medication. Another one-fifth have severe disease with life-threatening muscle weakness—swallowing difficulty may cause them to choke, for example, or they may need mechanical ventilation to help them breathe. “We see the latter in 15% to 20% of individuals at some point, especially early in the progression of the disease, when a patient can have what’s termed a ‘myasthenic crisis’ or exacerbation,” Dr. Nowak says.

What are researchers learning about the types of myasthenia gravis?

The latest knowledge about myasthenia gravis has changed how experts view the disease. “It turns out that within that whole population of myasthenia gravis patients, there are different autoantibodies that target different parts of the neuromuscular junction,” says O’Connor.

Two myasthenia gravis phenotypes are well known. In the most common type (an estimated 80%-85% of cases), the body’s immune system targets a protein on muscle cells required for muscle contraction called the acetylcholine receptor (AChR), weakening the muscle over time. About 15% of people with the condition don’t have detectable AChR in their blood, but about half of these will have autoantibodies to another protein called muscle-specific kinase (MuSK).

“If you were to meet patients in either of these two categories—AChR or MuSK—all of them would have muscle weakness and be diagnosed with myasthenia gravis,” O’Connor says. But the autoantibody subtypes separate them into two types of autoimmunity—and within each of those two groups of patients, there are variations that may affect responses to treatment, he adds.

“We’re trying to understand the mechanisms within these patient groups more deeply so we can understand why two different patients can have the same disease and autoantibodies, but one patient will respond remarkably well to a particular therapeutic and the other won’t. That’s where we’re headed,” says O’Connor.

What are the FDA-approved treatments for myasthenia gravis?

The following FDA-approved medications for generalized myasthenia gravis don’t cure the disease, but they may lessen the symptoms:

  • Eculizumab (brand name Soliris®). This medication, approved in 2017, blocks an immune mechanism called the complement system, which plays a key role in the disease. Eculizumab is given to patients in weekly intravenous (IV) infusions for four weeks, followed by maintenance doses every two weeks.
  • Efgartigimod (brand name Vyvgart®). This drug, approved in 2021, lowers the level of antibodies (and pathogenic autoantibodies) in the body below the threshold that allows the disease to remain active. It’s given in treatment cycles, each consisting of one IV infusion treatment a week for four weeks with a break between cycles.
  • Ravulizumab (brand name Ultomiris®). This treatment, approved in 2022, is the first long-acting complement inhibitor, and its mechanism of action is similar to eculizumab. After the first treatment, given by IV infusion, maintenance doses are given every eight weeks.
  • Rozanolixizumab-noli (brand name Rystiggo®). Approved in 2023, this is the first FDA-approved treatment for both anti-AChR and anti-MuSK antibody-positive myasthenia gravis. It targets a receptor that can stop harmful antibodies from being broken down by the cells’ natural waste clearance system. It’s administered by subcutaneous (under the skin) infusion once a week for six weeks.
  • Zilucoplan (brand name ZILBRYSQ®). This was also approved in 2023, and it’s the first myasthenia gravis therapy for self-administration by a daily injection. It works using a targeted mechanism to inhibit damage to the neuromuscular junction.

None of these medications are 100% effective for all patients, Dr. Nowak explains. “They are beneficial for about 60%-70% of patients, which leaves 30%-40% without any meaningful improvements,” Dr. Nowak says. But, he adds, many patients do well when the treatment is well-matched to their pathology.

For patients with myasthenia gravis characterized by the MuSK autoantibodies, another treatment called rituximab (brand name Rituxan®), also given by infusion, is an option, O’Connor adds.

Rituximab is approved to treat non-Hodgkin lymphoma and rheumatoid arthritis but is used off-label to suppress the production of abnormal antibodies in people with MuSK myasthenia gravis, he explains. “Rituximab depletes many of your B-cells [immune cells that play a role in the disease]. That medication doesn’t work well in some AChR patients, but it works remarkably well in MuSK patients,” O’Connor says. “When MuSK patients are treated with rituximab, often they will go into complete remission and require no additional immunotherapy for years. That’s something that wasn’t established a decade ago.”

“Rituximab has become a go-to option for many of our patients with MuSK Myasthenia gravis,” adds Dr. Nowak.

Are newer myasthenia gravis medications safer?

The newer medications have an established safety profile in myasthenia gravis, although there are still concerns—as there are with many medicines. For instance, patients taking eculizumab are at risk for developing life-threatening meningococcal infections, so anyone considering that medication must receive meningococcal vaccinations before their first dose.

By comparison, some older medications have led to serious complications, including an increased risk of cancer in young people who take them for decades. They’re also not used in individuals considering pregnancy, adds Dr. Nowak.

Corticosteroids, such as prednisone, used at significant doses for years predispose people to such conditions as bone loss, diabetes, high blood pressure, and obesity. “So, we tend to shy away from the long-term use of corticosteroids, especially in younger patients,” he says. “But, if a patient is older and only a small dose of prednisone is needed, this still might be the best and safest strategy when considering possible risks and benefits.”

Are there any non-drug treatments and/or self-care measures that can help with myasthenia gravis symptoms?

Physical activity and maintaining a healthy diet are important, explains Dr. Nowak. “Patients may get deconditioned due to weakness when myasthenia gravis is active, so we recommend physical therapy and general, light exercise and stretching to counter this,” says Dr. Nowak. “It may seem counterintuitive, but with this condition, the less you do—the less you will be able to do.”

That said, it’s important to listen to your body and work with your doctor, he adds.

How do you know if you have myasthenia gravis?

While symptoms like muscle weakness may be a clue that a person has myasthenia gravis, it can take years to diagnose in some cases; Dr. Nowak says many patients report a diagnosis delay of up to two years. This is partly because the condition can be confusing, starting with a mild symptom, such as a minor eyelid droop that comes and goes. “This is a rare disease, so someone could be seeing a primary care doctor who doesn’t find any specific abnormalities on an exam or consider that they could have myasthenia gravis,” says Dr. Nowak. “The condition can fluctuate in that individuals might be completely fine in the morning and develop double vision or slurred speech only at the end of the day. If a doctor is not looking for or familiar with myasthenia gravis, it may be hard to diagnose.”

A neurologist who suspects the condition will perform a physical and neurological examination. There are imaging studies, including brain imaging in some cases, since some symptoms of myasthenia gravis are similar to those of stroke or other brain conditions. There are also tests for eye muscle weakness and other specific electrodiagnostic tests, such as electromyography (EMG), which measures muscle response or electrical activity in response to a nerve’s stimulation of the muscle. A pulmonary function test can help in cases of possible myasthenic crisis. And blood tests can detect the presence of AChR or MuSK autoantibodies.

About 70% to 80% of myasthenia gravis patients will test positive for autoantibodies specific to the condition, explains Dr. Nowak, including whether a patient has AChR or MuSK autoantibodies. “Then, we have a group of people, about 20% to 25%, who are ‘seronegative,’ meaning that the autoantibody tests come back negative,” he adds. “That doesn’t mean that the individual doesn’t have autoimmune myasthenia gravis—it’s more a matter of the testing that we have available likely not picking up any autoantibodies.”

Seronegative results can be frustrating and lead to delays in diagnosis, Dr. Nowak says. But the clinical examination and electrodiagnostic testing can be sensitive and specific enough to confirm myasthenia gravis diagnosis in those patients, he adds.

How do doctors determine the best treatment for each patient?

“The Yale Medicine Myasthenia Gravis Program is very personalized, with an eye towards precision medicine health care,” Dr. Nowak says. “But it can be a journey.” A particular patient may need to try different medications, he adds. “There may be fine-tuning in terms of the treatments that we offer each individual.” For example, people can develop myasthenia gravis at any age, from younger than 10 to older than 90, and the medications can have different effects, depending on a person’s age, he explains. “The treatment strategies we use for younger patients might not be the same as those we use for older people. The same can be said for those with mild versus severe disease.”

But “myasthenia gravis is a treatable disease for most patients once we find the right treatment strategy for them,” Dr. Nowak says, and the medications are likely helping more patients to achieve better outcomes, he adds.

Close and careful follow-up care with a neurologist is essential. “The first two to three years from initial symptom onset is sometimes the most challenging and critical because patients can progress from mild to very severe disease during this time,” he says.

Patients may go into remission or have disease relapse, and even the mechanisms driving their disease might change, so the therapeutics used may change over time, too. While there are more available treatment options, we still do not have a way to predict which treatments will work the best for which patients. “This represents a knowledge gap we’re hoping to address,” Dr. Nowak says.

Is surgery used as a treatment for myasthenia gravis?

A surgery called a thymectomy (removal of the thymus gland) can lessen symptoms and the need for medication. An international clinical trial published in the New England Journal of Medicine in 2016 addressed a question doctors have sought to clarify for decades about the efficacy of thymectomy in patients without thymoma, “providing evidence that supported the use of thymectomy for improving clinical outcomes and reducing the need for immunosuppressive therapy in patients with myasthenia gravis,” according to the authors. In the study, thymectomy was associated with a more favorable outcome than treatment with prednisone alone.

Those diagnosed with myasthenia gravis in their 20s, 30s, and 40s, for instance, sometimes have an enlarged thymus gland, called thymic hyperplasia, explains Dr. Nowak. “For younger patients with AChR myasthenia gravis, we recommend elective thymectomy based on available evidence,” he says.

In about 10% of patients, a benign tumor in the thymus gland (called a thymoma) is detected, and in those cases, a thymectomy is typically needed. That’s why patients diagnosed with myasthenia gravis are given a CT scan of the chest as part of their diagnostic workup. When a thymoma is diagnosed, surgeons can remove it and the thymus tissue surgically—commonly using a minimally invasive approach.

“As we age, the thymus shrinks, so the utility of an elective thymectomy in individuals without a thymoma later in life is not established and thought to not be beneficial,” says Dr. Nowak.

How might myasthenia gravis treatment change in the future?

Both O’Connor and Dr. Nowak hope to see a continuing paradigm shift in myasthenia gravis treatment in the next seven to ten years based on research. Doctors are already moving away from the traditional practice of treating myasthenia gravis symptomatically and then giving patients global immunosuppressive therapies without a specific focus, Dr. Nowak explains. The new therapeutics target specific parts of the immune system, he adds.

Still, “all of these therapeutics, while effective, are only eliminating the immunological activity,” O’Connor says, explaining that researchers want to target the source of that irregular activity. “The body is not supposed to make antibodies to itself. Your system has built-in checkpoints that prohibit that from happening, and they’re not functioning properly in these patients—or in any patient with an autoimmune disease.”

Fixing the defect or defects that allow the autoantibodies to be produced would be closer to a cure and eliminate the need for chronic therapy, he adds.

While rare diseases tend not to get as much attention and funding as such common ones as diabetes and heart disease, myasthenia gravis research got a boost from a 2019 NIH grant to develop the Myasthenia Gravis Rare Disease Research Network or MGNet.

Yale is one of only a handful of centers in the United States that is part of MGNet, a consortium of academic medical centers and others working together to improve the fundamental understanding of and enhance therapeutic development for myasthenia gravis.

“One of the main projects is a natural history study, EXPLORE-MG2, where we’re following newly diagnosed patients for a minimum of 2 years in an effort to obtain a deeper characterization of the disease over time. We are not only monitoring patients clinically but also exploring how their immune system changes from diagnosis to six months, one year, and two years down the line,” Dr. Nowak says. “Research efforts such as these are the foundation to a path toward precision medicine health care, and hopefully will help us not only identify but validate treatment-predictive biomarkers.”

For most patients, myasthenia gravis can now be considered a chronic disease that is treatable with the right individual treatment strategy—making the condition much less threatening than it was even five to 10 years ago, Dr. Nowak explains.

“Soliris, the first of the new drugs, was transformative in that for many of those patients, we had exhausted our treatment options, and when this new medication became available, their myasthenia gravis could be managed a lot better,” he says. “Helping patients with myasthenia gravis who need more options is a driving force behind research to find new treatments with improved outcomes.”

As more options become available, we will need to be equally focused on understanding which strategies are best for which patients, he adds.

https://www.yalemedicine.org/news/myasthenia-gravis-new-drugs-individualized-treatment