What is SMA? Key signs to look out for in babies as Jesy Nelson highlights life-changing condition - Pop star Jesy Nelson has ...

Gemma Biotherapeutics ("GEMMABio"), a clinical‑stage, global, genetic medicines company, today announced the presentation of preclinical data supporting candidate declaration for GB703, a novel, ...

Key signs of spinal muscular atrophy include severe muscle weakness, difficulty breathing or swallowing, and missing ...

Myotonic dystrophy type 1 (DM1) is a genetic disease characterized by progressive muscular weakness. There is currently no treatment despite many recent efforts. But now researchers from Japan may ...

A new mouse model mimicking the liver symptoms of myotonic dystrophy type 1 -- the most prevalent form of adult-onset muscular dystrophy -- provides insight into why patients develop fatty liver ...

Pop star Jesy Nelson has campaigned for screening to be rolled out after her twins were diagnosed with the condition ...

Using brain organoids, researchers discover mutational commonalities between muscular dystrophy type 1 and Rett syndrome, suggesting the potential of a similar treatment for both. Myotonic dystrophy ...

Entrada Therapeutics Inc ( (TRDA)) has issued an announcement. On May 7, 2026, Entrada Therapeutics reported first-quarter 2026 results and unveiled positive topline data from Cohort 1 of its Phase ...

In a preprint uploaded to the Research Square* server, researchers used a combination of in vivo mice models and next-generation sequencing techniques to elucidate the pathomechanistic connection ...

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 ...