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Newborn heel prick test and lood puncture, Taking a Heel Blood Sample From Newborn Baby

Families Are Waiting

A recent 60 Minutes segment examining the cost of gene therapy put a national spotlight on a question families living with rare disease confront every day: how can lifesaving medical breakthroughs exist if they remain out of reach for the people who need them? The public reaction to the multimillion-dollar price tags for gene therapies…

Research Lab Scientist Using Computer. Medical Science

Everything You Need to Know About Clinical Trials for Neuromuscular Diseases

Clinical trials are key to advancing research for neuromuscular disease treatment. Here’s what you need to know why to get involved and how to enroll.

A Year Built Together: Reflections on 2025 and the Road Ahead

Stepping into this role in such a historic year has been humbling and inspiring. This is my first End of Year message as MDA’s President and CEO, and I want to begin by thanking you. Our progress in 2025 was driven by the partnership we share across this entire community of families, clinicians, researchers, volunteers,…

3D rendering of a biotinylated antibody linked to BODIPY molecules or bright green. This technique allows to visualize and track the antibody for targeted applications in medical research.

Simply Stated: Understanding Idiopathic Inflammatory Myopathies

The idiopathic inflammatory myopathies (IIMs) are a group of rare autoimmune muscle diseases that include dermatomyositis, polymyositis, immune-mediated necrotizing myopathy (IMNM), antisynthetase syndrome, and inclusion body myositis (IBM). These disorders cause progressive muscle weakness, can affect multiple organs, and often impact quality of life. Together, the IIMs affect an estimated 2 to 25 per 100,000…

Clinical Trial Alert Myotonic Dystrophy Type 1

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) to treat DM1. DM1 is caused by a defect in the DMPK gene that leads to the production of abnormally long DMPK RNA transcripts, which…

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 heard by lawmakers. As we prepare to enter 2026, we reflect on the remarkable work of MDA’s advocacy community this year. The fight to protect…

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 or HINALEA 2) to assess the safety and effectiveness of risdiplam (Evrysdi) when administered as an early intervention or who experienced a plateau or decline…

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 investigational therapy DYNE-101 to treat DM1. DM1 is caused by a defect in the DMPK gene that leads to the production of abnormally long DMPK…

Colorful figurines on the dark surface. Diversity and inclusion concept.

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 opportunity to live independently. The movement brought forth monumental change and opened the door for conversations that challenged stereotypes and stigmatized language. At that time,…

Disabled student in class room working with laptop.

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.

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 members of the neuromuscular disease community about topics that matter to them and to the larger community of individuals with disabilities.

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Closeup of Christie West and her 28-year-old son, Zach, smiling at the camera.

Health

The Hidden Power of Self-Care in Caregiving

By Rene Ryan | November 14, 2025

Action shot of Jake Saxton pumping his left fist with a triumphant look on his face and his mouth open in a yell. He is in a power wheelchair wearing a hockey jersey and knee and shin guards, with a power hockey stick resting in his right hand.

Inclusion

Meet Our 2025 MDA Quest Photo Contest Winners

By MDA Staff | November 14, 2025

Graduate student Naomi Hess sitting in a power wheelchair in front of a Northeastern University sign in Boston.

Personal Stories

A Lifetime of Learning: From College to a Career in Disability Advocacy

By Naomi Hess | November 14, 2025

Illustration of a woman sitting at a computer desk, surrounded by charts and graphs, a calculator, and money symbols.

Independence

New Year, New Financial Plan: Tips to Get Your Budget Back On Track

By Maggie Callahan | November 14, 2025

Illustration of a human brain, including the bulb-like structure in the lower part of the brain

Science + Research

Understanding Spinal-Bulbar Muscular Atrophy

By Matt Schur | November 14, 2025

Closeup of Eric Arnold, a light-skinned man with a closely trimmed brown beard and wire-rimmed glasses, wearing a baseball cap that says WVU Alumni.

Inclusion

From Classroom to Courtroom: How Eric Arnold Navigated Accessibility in His Education and Career

By Rebecca Hume | November 14, 2025

By Steve Wright | December 15, 2025

QUEST PODCAST

The Quest podcast, proudly presented by the Muscular Dystrophy Association, is part of the Quest family of content. Hosted by Quest Editor-in-Chief, motivational speaker and writer Mindy Henderson.

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Episode 58- Wrapping Up 2025 with Ira and Lily

December 1, 2025

In this Quest Podcast episode, we chat with Muscular Dystrophy Association’s National Ambassadors, Lily S. and Ira Walker. Lily is a dedicated advocate finishing her first year as a National Ambassador. She shares her journey, why she believes it is important to advocate for yourself and others, and what she has learned along the way….

Episode 57- Voices of Inclusion: Celebrating NDEAM with Disability:IN

October 21, 2025

October is National Disability Employment Month (NDEAM). In this month’s episode of the Quest podcast, we dive into accessibility and inclusion in the workforce with Russell Shaffer, Executive Vice President of Strategy & Programs at Disability:IN. Drawing on his lived experience of vision loss and his years of working in corporate diversity, equity, and inclusion,…

Episode 56- Precision Medicine: Mapping the Genetic Code for New Treatments

September 24, 2025

In this Quest Podcast episode, we chat with Dr. Stephan Züchner, Dr. Conrad “Chris” Weihl, and the Interim Chief Research Officer of the Muscular Dystrophy Association, Dr. Angela Lek. Leaders in the field of genetic mapping, all three have devoted their time and expertise to research and treatments for neuromuscular diseases. Their goal is to…

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