DM1

Disease

Related News

Avidity's Myotonic Dystrophy Drug Shows Promising Phase 1/2 Trial Results

Mar 26, 2026

Avidity Biosciences announced promising Phase 1/2 trial results for its myotonic dystrophy type 1 drug delpacibart etedesiran, showing 40% reduction in DMPK mRNA and functional improvements. The therapy has received multiple regulatory designations and is now in Phase 3 testing with data expected in late 2026. The company's stock has surged 128% over the past year despite reporting a net loss of approximately $550 million.

Orphan Drug Designation Drives Rare Disease Therapy Development Across Global Markets

Mar 15, 2026

Regulatory incentives for rare disease treatments, including market exclusivity and development subsidies, are spurring pharmaceutical innovation across the US, Europe, Japan, and Australia, with multiple companies advancing therapies for conditions affecting limited patient populations.

Dyne Therapeutics Advances Neuromuscular Disease Programs Toward Commercialization

Mar 06, 2026

Dyne Therapeutics is preparing a U.S. Accelerated Approval submission for z-rostudirsen in Duchenne muscular dystrophy and gearing up for a Phase 3 trial of z-basivarsen in myotonic dystrophy, while scaling operations to support potential commercialization.

FDA Places Partial Clinical Hold on PepGen's FREEDOM2-DM1 Trial

Mar 05, 2026

The FDA has placed a partial clinical hold on PepGen's Phase 2 FREEDOM2-DM1 trial for myotonic dystrophy type 1, citing questions about preclinical pharmacology and toxicology studies. The company is submitting additional analyses to address the FDA's concerns.

Del-desiran Shows Muscle Delivery and Splicing Improvements in Phase 1/2 DM1 Trial

Feb 19, 2026

Phase 1/2 MARINA trial results published in NEJM show del-desiran achieved approximately 40% reduction in toxic DMPK mRNA in muscle tissue and improvements in myotonia and functional measures in myotonic dystrophy type 1 patients, with acceptable safety profile.

Dyne Therapeutics Receives Orphan Drug Designation in Japan for DM1 Treatment

Feb 17, 2026

Japan's Ministry of Health, Labour and Welfare granted Orphan Drug designation to Dyne Therapeutics' investigational treatment zeleciment-basivarsen for myotonic muscular dystrophy type 1, adding to existing designations in the U.S. and Europe.

Vertex Reports Q4 2025 Revenue of $3.19B, Issues 2026 Guidance of $12.95-13.1B

Feb 13, 2026

Vertex Pharmaceuticals reported fourth quarter 2025 revenue of $3.19 billion, up 10% year-over-year, and provided full year 2026 revenue guidance of $12.95 billion to $13.1 billion with over $500 million expected from non-CF products.

Related Clinical Trials

NCT ID	Title	Status	Phase
NCT07505342	Remote Assessments and Genetic Determinants of Myotonic Dystrophy	RECRUITING
NCT07486934	Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1	RECRUITING	PHASE3
NCT07385443	The Spanish National Registry for Myotonic Dystrophy Type 1	RECRUITING
NCT07008469	Global Open-Label Extension Study of Del-desiran for the Treatment of DM1	ENROLLING_BY_INVITATION	PHASE3
NCT06411288	Global Study of Del-desiran for the Treatment of DM1	ACTIVE_NOT_RECRUITING	PHASE3
NCT05479981	Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients	COMPLETED	PHASE2
NCT05027269	Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients	COMPLETED	PHASE1/PHASE2
NCT03981575	Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)	RECRUITING
NCT02084407	Induction of Pluripotent Stem Cells From Human Fibroblasts of DM1 Patients	COMPLETED	NA