Archives
Clinical Research Alert: Phase 1 Study of Gene Therapy in Individuals with Myotonic Dystrophy Type 1
Researchers at Sanofi are seeking individuals with non-congenital myotonic dystrophy type 1 (DM1) to participate in a phase 1/2 clinical trial to evaluate the safety of an investigational gene therapy (SAR446268)…
Reflecting on 2025’s Advocacy Accomplishments
The last 12 months have been full of twists and turns in Washington D.C., but through all the unexpected events, MDA’s advocates remained steadfast and ensured that their voices were…
Tags: Accessible Air Travel, Advocacy Updates, Community, MDA Advocacy, MDA Hill Day, MDA impact, Resources
Clinical Research Alert: Two Phase 4 Studies of Risdiplam in Pediatric SMA Patients Following Gene Therapy
Researchers at Genentech Inc. are seeking pediatric patients previously treated with gene therapy (onasemnogene abeparvovec) for spinal muscular atrophy (SMA) to participate in either of the two interventional studies (HINALEA 1…
Clinical Research Alert: Phase 1/2 Study of DYNE-101 in Individuals with Myotonic Dystrophy Type 1
Researchers at Dyne Therapeutics, Inc. are seeking individuals with myotonic dystrophy type 1 (DM1) to participate in a phase 1/2 clinical trial (ACHIEVE) to evaluate the safety and efficacy of the…
A Shift in Perspective: Person-First Language and Identity-First Language
When the disability rights movement began in the 1970s, the disability community started forging the path for those living with disabilities to have access to equal rights, inclusion, and the…
Tags: Ambassadors, Community
Navigating College Scholarships and Financial Assistance with a Disability
Tips for finding funding—from scholarships to financial aid—to help students with disabilities start and finish college.
Tags: College, Education, Featured Content, Finance, MDA College Scholarships, Resources, Transitions, Young Adults
Insights by Ira: Mission Complete
Just four days prior to my inaugural public engagement as National Ambassador in 2024, I began not feeling well. Confronted with this situation, I was faced with two options: notify…
Tags: Ambassador Guest Blog, Ambassadors, Community, Insights by Ira, Ira, MDA Ambassador Program, MDA National Ambassador Blog, MDA National Ambassadors, Mental Health, Relationships
In Case You Missed It…
Quest Media is an innovative, adaptive lifestyle platform from MDA. With the power of this platform, we foster awareness and empowerment and have important conversations with experts, thought leaders, and…
Tags: Ambassador Guest Blog, Ambassadors, Caregiving, Community, Drug Approval, Drug Development, Employment, Finance, Genetic Testing, Healthcare, In Case You Missed It, Ira, Lily, Parenting, Research
Clinical Research Alert: Phase 1b Study of SRD-001 in Adults with DMD-Related Cardiomyopathy
Researchers at Sardocor Corp are seeking adults with cardiomyopathy (heart disease) secondary to Duchenne muscular dystrophy (DMD) to participate in a phase 1b clinical trial to evaluate the safety and explore…
Holiday Gift Wrapping Made Easier: Accessible Tips & Tricks
As the holidays approach, ‘tis the season for holiday hacks and tips. One key (and sometimes daunting) task on many people’s holiday to-do list after the busy hustle and bustle…
Tags: Community