Medically reviewed by Smita Patel, DO Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed ...

研究人员首次发现，人类细胞自带一套精密的"应急修复系统"，当检测到关键基因故障时，会自动激活"替补基因"展开修复。更令人振奋的是，这项机制可被人工干预精确调控，为DMD乃至6000多种单基因遗传病带来 ...

Muscular dystrophy is a disease caused by a genetic mutation. The muscles of muscular dystrophy patients weaken.. | Genetics ...

Utrophin increase in muscle cells after transcriptional adaptation normalises cell function in Duchenne muscular dystrophy ...

Solid Biosciences’ gene therapy uses a shortened version of the dystrophin protein, which is engineered to mimic the key ...

Duchenne muscular dystrophy (DMD) is a rare hereditary disease that is associated with progressive muscle wasting. The ...

People with Duchenne or Becker muscular dystrophy who had more siblings typically report being better prepared to transition to adult life, a study says.

Managed Healthcare Executive provides C-suite executives at health plans and provider organizations with news, analysis, ...

Around 13% of DMD patients respond to exon 51 skipping, but PepGen reckons other drugs in its pipeline will address mutations that are central to around 35% of all cases of the disease.

Abstract Title: Safety and Efficacy of DYNE-101 in Adults with DM1: Phase 1/2 ACHIEVE Trial Data Date and Time: Wednesday, March 19, at 12:30-12:45 p.m. CT Presenter: James Lilleker M.D., Neurologist, ...

The Swiss biopharma company said the deal with the German National Association of Statutory Health Insurance Funds (GKV-SV) ...