The data showed reduced difficulties in standing, walking and running that were statistically significant, the company said.

While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

Pratteln, Switzerland, January 16, 2025 – Santhera Pharmaceuticals (SIX: SANN) announces that the National Institute for Health and Care Excellence (NICE) has issued positive Final Guidance that ...

Altruism values for treatments of rare, severe pediatric diseases have not been estimated. This study found the altruism value for a hypothetical new Duchenne muscular dystrophy treatment to be $80 ...

Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant muscle disease with a prevalence of 1/10,000–1/20,000 (Lunt and Harper, 1991; Deenen et al., 2014; Tihaya et al., 2023). It is ...

Dysferlin-deficient muscular dystrophy is a devastating and untreatable disease. Using Cas9, the authors restored dysferlin in muscle stem cells from patients ex vivo and show proof-of-concept for ...

Arsenal goalscorer Ethan Nwaneri was substituted at half-time against Brighton & Hove Albion because of a muscular injury, Mikel Arteta has said. The 17-year-old opened the scoring for Arsenal in ...

offering new pathways for understanding and treating Duchenne Muscular Dystrophy (DMD). DMD, a severe genetic disorder that causes muscle weakness and shortens lifespans, arises from mutations in ...

offering new pathways for understanding and treating Duchenne Muscular Dystrophy (DMD). A groundbreaking study has shed light on the complex interactions between dystrophin, a protein critical to ...