Archives
How Small Endeavors Make a Big Impact
MDA’s mission to fund groundbreaking research for neuromuscular disease treatment and provide families with the highest quality care would not be possible without the generous donation of time and resources…
Tags: Community, Fill the Boot, Muscle Walk, Shamrocks, Summer Camp, Volunteers, Year of the Volunteer
Make Sure You Don’t Lose Your Medicaid Coverage
Many Americans, including those living with a neuromuscular disease, rely on Medicaid to access vital medical care, especially during the COVID-19 pandemic. However, as the federal Public Health Emergency (PHE),…
Clinical Trial Alert: Phase 2 Study of ALXN2050 in Adults with Generalized Myasthenia Gravis (gMG)
Researchers at Alexion AZ Rare Disease are seeking adults living with generalized myasthenia gravis (gMG) to participate in a phase 2 clinical trial (ExpanD Study) to evaluate the safety and efficacy…
For Ukrainians with Rare Diseases, Life is Still a Struggle
February 28 marked Rare Disease Day — an annual event calling attention to the 300 million people worldwide who live with one or more of the 7,000 illnesses classified as…
Tags: Community, Healthcare
2023 MDA Clinical & Scientific Conference: Abstract Library & Agenda
The 2023 MDA Clinical & Scientific Conference is just around the corner. With 32 sessions, 184 speakers, 61 exhibitors and patient advocacy organizations and 14 Industry Forums, this year’s conference…
FDA Approves Reata’s SKYCLARYS for Treatment of FA
On Feb. 28, the US Food and Drug Administration (FDA) granted approval to omaveloxolone (SKYCLARYS™) for the treatment of Friedreich’s ataxia (FA) in adults and adolescents aged 16 years and…
Tags: Drug Approval, Research Advances
Self-Defense With a Disability
By thinking about personal safety and learning about self-defense with a disability, you can feel more protected and confident outside your home.
Tags: Staying Active, Young Adults
Simply Stated: Research Updates in Facioscapulohumeral Muscular Dystrophy (FSHD)
Facioscapulohumeral muscular dystrophy (FSHD) is a rare genetic disorder that affects the muscles of the face (facio), shoulders (scapulo), and upper arms (humeral). It is one of the most common…
Tags: Healthcare, Research, Simply Stated
What About My Disease? Why a New Treatment for One Neuromuscular Disease Is Good News for All
New therapies and research advances for some neuromuscular diseases are paving the way for developing new treatments for other rare diseases.
Tags: Antisense Oligonucleotide, Clinical Trials, Drug Development, Featured Content, Gene Therapy, Innovation, Research
MDA National Ambassador Blog-Letters From Leah: What I’d Like Parents to Know About Being a Teenager with a Disability
Leah is an MDA National Ambassador who lives with a rare form of neuromuscular disease. Dear Parents, Hi, my name is Leah and I am 15 years old. I live…
Tags: Ambassadors, Community, Leah, Letters From Leah, Parenting, Young Adults