The name myasthenia gravis comes from Greek and Latin words meaning “grave muscle weakness.” This rare autoimmune disease occurs in all races, male or female, and at any age.
Living with myasthenia gravis (MG) can be a challenge. When severe, it can result in difficulty chewing, smiling, swallowing, talking, or breathing. MG is not thought to be directly inherited, however it does occasionally occur in more than one member of the same family. Nancy Law, Chief Executive of the Myasthenia Gravis Foundation of America (MGFA) shares more about what her organization is doing to help find a cure for MG.
The National MG Conference will be held in Kansas City, MO April 15-17, 2018. PRA’s Lisa Dilworth, Director of Therapeutic Expertise will be moderating the session: Understanding Clinical Trials and How They Help Patients. She is also organizing a walk at the conference. Visit her PRA Cares team page. Lisa also interviewed her mom who has MG; read more here.
Can you tell us about the Myasthenia Gravis Foundation of America and how you help patients and their families?
The Myasthenia Gravis Foundation of America (MGFA) is the only national voluntary health organization dedicated solely to myasthenia gravis and related disorders. The mission of the MGFA is to facilitate the timely diagnosis and optimal care of individuals affected by myasthenia gravis and closely related disorders and to improve their lives through programs of patient services, public information, medical research, professional education, advocacy, and patient care. Our vision is a world without myasthenia gravis.
Can you tell us about your MG patient registry? How will it help with research and treatment?
MGFA launched the MG Patient Registry to help expand knowledge about MG and move us closer to improved treatments and a cure. Through the Registry, the MG community will help identify how each of us was diagnosed, how we are being treated, our insurance challenges, and how we are feeling.
Myasthenia gravis is a rare disease and every patient is like a “snowflake” with a unique experience of MG. Having a significant number of registry participants is essential to getting a more complete picture of the MG community and what patients face.
By making a patient community more accessible and understandable, a patient registry and its bounty of information can encourage pharmaceutical developers to pursue drug discovery for a disease.
Our registry vision is to:
- Offer a confidential means for patients to provide information that will be useful to medical/research communities
- Provide a system for investigators to gauge the potential for trial recruitment and to communicate with patients directly about potential research investigations
- Educate patients, caregivers, non-expert health care providers, and funding entities
How has MGFA supported research efforts? What breakthroughs are you most encouraged by?
The research committee of the MGFA is committed to supporting research that will ultimately improve the lives of patients with myasthenia gravis and related neuromuscular junction disorders. The committee has identified four broad research priorities of unmet need in the field:
- Early diagnosis
- Predictors of clinical outcome
- Predictors of response to immunosuppressive therapy
- Biomarkers for use in clinical trials
- Mechanisms of disease
- Basic mechanisms of disease
- How loss of self-tolerance is sustained throughout the disease course
- Therapeutic strategies
- Development of new therapeutic targets
- Targeted therapies that hopefully prevent widespread immunosuppression and off-target side effects
- Optimizing treatment strategies with existing therapies
- Improving patient outcomes
- The patient perspective on their disease, its impact on their daily life, and treatment considerations that are most important to them
- Collateral effects of MG and its treatment such as associated medical conditions, treatment related side effects, and financial considerations
Key breakthroughs in the past few years include:
- International Treatment Consensus Guidelines
- Results of 6-year thymectomy study
- First new treatment approved for MG since 1955
What is the MyMG App?
“My MG” is a software application designed to run on smartphones, tablet computers, and other mobile devices.
Using the “myMG” app to record MG symptoms and other notes enables users to have a more interactive dialogue with doctors. They can record symptoms and how they affect their daily life. The app also allows users to record any notes that may explain changes in their symptoms (change in meds, forgetting meds, ran a marathon, for example).
A charts tab enables users to see how their survey results change over time. This is information they can share with their doctor if they choose.
What is the theme for this year’s National Conference? What are your goals?
The conference theme for this year is “Be the Change!” Goals include:
- Bring members of the MG Community together to learn and exchange ideas
- Provide education from more than 70 presenters and speakers who are top experts in their field—and extending the opportunity to learn to the entire MG community by recording all general sessions and some breakouts
- Inspire activism and engagement from MG community members
- Help people feel empowered and able to manage the challenges of MG in their lives
MGFA organizes a number of walks across the US to rally the MG community, help raise awareness of MG and the challenges of living with it, and to fund critical research and service programs. More information can be found at www.MGWalk.org
This week’s issue of Industry Watch is focused on proposed changes to US lung cancer screening guidelines which will hopefully result in increased…
Rare Diseases Newsletter Volume 16, April 2020
Decentralized Trials: Reshaping the Rare Disease Landscape
Clinical drug development is moving away from traditional approaches that rely on rare disease patients to travel long distances to clinical sites…