Avidity's delpacibart etedesiran (del-desiran) was granted orphan drug status in Japan as a treatment for myotonic dystrophy ...

Women with high-risk HER2-positive breast cancer lived significantly longer with adjuvant trastuzumab emtansine (T-DM1, Kadcyla), according to long-term follow-up of a large clinical trial.

Avidity Biosciences receives Japan MHLW’s orphan drug status for delpacibart etedesiran to treat myotonic dystrophy type 1: San Diego Thursday, April 10, 2025, 13:00 Hrs [IST] A ...

Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics ...

See what stocks are receiving strong buy ratings from top-rated analysts. Filter, analyze, and streamline your search for investment opportunities with TipRanks’ Stock Screener. The latest ...

Myotonic Dystrophy Type 1 (DM1) is the most common adult-onset form of muscular dystrophy and a condition that severely affects multiple organs including skeletal muscle, heart, brain and the ...

How would you summarize your study for a lay audience? Myotonic dystrophy type 1 (DM1), a genetic disorder, shares overlapping features with chronic kidney disease, including progressive muscle ...

We also investigate the pathogenic mechanisms of myotonic dystrophy, type 1 (DM1), an autosomal dominant neuromuscular disorder affecting multiple tissues including muscle, heart and the central ...

In recent years, a growing understanding of myotonic dystrophy type 1 (DM1)—an often fatal, devastating, rare neuromuscular disorder that causes muscle weakness and other life-limiting ...

PepGen halts its Phase 2 CONNECT2 trial for PGN-EDO51 in DMD to assess 10 mg/kg cohort data, while advancing FREEDOM-DM1 studies in myotonic dystrophy. PepGen shares are trading higher by 90% ...