December 2025

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…

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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…

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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…

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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,…

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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…

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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…

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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.…

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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 newborn screening across all states. PPMD has been at the forefront of efforts to advocate for Newborn Screening for more…

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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…

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Like many high school students, students with neuromuscular diseases may be looking at college as their next step. But with college tuition costs continuing to spiral, according to U.S. News & World Report, you might be wondering how you’ll afford it. The key may be combining scholarships, grants, and other financial aid programs and benefits.…

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