News

I’m feeling funny today — humorous, in fact. Brimming with sarcasm and a splash of laughter, today feels like a cocktail, served over ice and garnished with a tiny umbrella and an extra-long, twisty straw. The main ingredient? Wit, with a dash of mischief. Why? Why not. Every so often, I revisit my past columns The post Life is so much easier with a sense of humor appeared first on Muscular Dystrophy News.

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… The post Simply Stated: Understanding Idiopathic Inflammatory Myopathies appeared first on Quest | Muscular Dystrophy Association.

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… The post Clinical Research Alert: Phase 1 Study of Gene Therapy in Individuals with Myotonic Dystrophy Type 1 appeared first on Quest | Muscular Dystrophy Association.

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… The post Reflecting on 2025’s Advocacy Accomplishments appeared first on Quest | Muscular Dystrophy Association.

Every newborn in the United States should now be screened for Duchenne muscular dystrophy (DMD), according to a recommendation from the U.S. Department of Health and Human Services (HHS). This federal decision marks a turning point for rare disease advocacy, potentially ending the “diagnostic odyssey” that many families face when seeking answers for their children’s The post Advocates cheer federal push to test every newborn for Duchenne appeared first on Muscular Dystrophy News.

This column has followed me through a year that has asked a lot of my body and my heart. Looking back at what I’ve penned this year, 2025 reads like a bittersweet journal of my relationship with Duchenne muscular dystrophy (DMD), one where grief, growth, pride, and fatigue all took center stage at various points. The post Reflecting on another year of living with DMD appeared first on Muscular Dystrophy News.

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… The post Clinical Research Alert: Two Phase 4 Studies of Risdiplam in Pediatric SMA Patients Following Gene Therapy appeared first on Quest | Muscular Dystrophy Association.

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… The post Clinical Research Alert: Phase 1/2 Study of DYNE-101 in Individuals with Myotonic Dystrophy Type 1 appeared first on Quest | Muscular Dystrophy Association.

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,… The post A Shift in Perspective: Person-First Language and Identity-First Language appeared first on Quest | Muscular Dystrophy Association.

Today marks a monumental victory for the Duchenne community. The U.S. Department of Health and Human Services (HHS) has officially added Duchenne to the Recommended Uniform Screening Panel (RUSP)—the national list of conditions recommended for… The post Historic Victory: Duchenne Added to the Recommended Uniform Screening Panel (RUSP) appeared first on Parent Project Muscular Dystrophy.

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to (Z)-endoxifen for Duchenne muscular dystrophy (DMD), a step that could allow its developer, Atossa Therapeutics, to qualify for a future priority review benefit for another experimental treatment in its pipeline. “This designation is an important regulatory milestone for Atossa, and we believe a The post FDA designation highlights new treatment approach for DMD appeared first on Muscular Dystrophy News.

Tips for finding funding—from scholarships to financial aid—to help students with disabilities start and finish college. The post Navigating College Scholarships and Financial Assistance with a Disability appeared first on Quest | Muscular Dystrophy Association.

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 my MDA contact regarding my potential absence from the highly anticipated event or adhere to my established practice of persevering through challenges in order to… The post Insights by Ira: Mission Complete appeared first on Quest | Muscular Dystrophy Association.

Dyne Therapeutics said it plans to ask the U.S. Food and Drug Administration (FDA) to approve DYNE-251, its exon-skipping therapy for people with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping, after a Phase 1/2 trial showed encouraging results. Data from the registrational expansion cohort of the DELIVER trial indicated that DYNE-251 is capable The post Dyne, citing positive trial data, to ask FDA to approve DMD therapy appeared first on Muscular Dystrophy News.

Advent is the beginning of the church year for Christians. What better way to start the year than with a season of hope? During Advent, Christians prepare for the coming of Jesus, whom we believe is the incarnation of God. He entered our world, becoming human, when he was born in Bethlehem. It’s no accident The post My hope for the FSHD community this Advent season appeared first on Muscular Dystrophy News.

In the Phase 3 HOPE-3 clinical trial, the experimental cell therapy deramiocel outperformed a placebo at improving arm function and heart health in people with Duchenne muscular dystrophy (DMD), according to top-line results announced by its developer, Capricor Therapeutics. Capricor has said it plans to use the HOPE-3 data as a basis to resubmit its The post New trial results highlight gains in arm and heart function for DMD appeared first on Muscular Dystrophy News.

Words matter. For that reason, I’d like to share my thoughts on language, chronic illness, and how words shape societal views. I believe the way we use language can improve how patients living with rare conditions are seen, understood, and treated by the world at large. I’ve lived with limb-girdle muscular dystrophy all my life The post Finding the right words to describe our life experiences appeared first on Muscular Dystrophy News.

Dyne Therapeutics has announced positive topline results from its Registrational Expansion Cohort (REC) of the Phase 1/2 DELIVER trial of zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) in people living with Duchenne who are amenable… The post Dyne Therapeutics Announces Positive Topline Results from Phase 1/2 DELIVER Trial of Z-Rostudirsen in Duchenne appeared first on Parent Project Muscular Dystrophy.

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. With so many valuable… The post In Case You Missed It… appeared first on Quest | Muscular Dystrophy Association.

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 the efficacy of the investigational gene therapy SRD-001 (AAV1/SERCA2a). In heart failure, the body does not make enough of the SERCA2a protein, which helps heart… The post Clinical Research Alert: Phase 1b Study of SRD-001 in Adults with DMD-Related Cardiomyopathy appeared first on Quest | Muscular Dystrophy Association.

For more than three decades, Parent Project Muscular Dystrophy (PPMD) has been committed to bringing therapies closer to, and improving care for, every person living with Duchenne and Becker muscular dystrophy. The Duchenne and Becker… The post Honoring Every Journey in Duchenne and Becker: Advancing Therapies, Access, and Care For All appeared first on Parent Project Muscular Dystrophy.

Navigating the holidays creates some logistical problems for my family. It’s not because there are nine of us. We might be a big family, but if we can all fit in our average-sized, ranch-style home, then we can fit in most homes. Having three power wheelchair users in the family does make getting into many houses The post Planning a ‘Krank’ Christmas and getaway with accessibility in mind appeared first on Muscular Dystrophy News.

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 of shopping for presents is: gift wrapping. For those living with neuromuscular disease, limited dexterity and/or range of motion and muscle weakness and fatigue can… The post Holiday Gift Wrapping Made Easier: Accessible Tips & Tricks appeared first on Quest | Muscular Dystrophy Association.

Progress in neuromuscular research has always depended on collaboration — scientists, families, advocates, and organizations uniting to accelerate the path to treatments. This spirit is at the heart of the Muscular Dystrophy Association’s latest announcement: nearly $2 million in new collaborative research grants awarded with seven partner organizations to advance breakthroughs across ALS, congenital myopathies,… The post Progress Through Partnership: MDA’s Collaborative Research Grants Drive Neuromuscular Science Forward appeared first on Quest | Muscular Dystrophy Association.

Upsher-Smith Laboratories is launching a new corticosteroid treatment for people with Duchenne muscular dystrophy (DMD) in the U.S. ages 5 and older. The therapy, branded Kymbee, is an oral tablet formulation of deflazacort, a corticosteroid available for DMD patients under brand names including Emflaza. Patients taking Kymbee will have access to Upsher-Smith’s Promise of Support The post DMD treatment Kymbee launches with support program for patients appeared first on Muscular Dystrophy News.