مقالات به زبان انگلیسی

A Gene Therapy Appears To Replace Missing Protein In Muscular Dystrophy Patients

نوشته شده توسط forbes.com در .

Jun 19, 2018

An experimental gene therapy appeared to dramatically increase the production of a muscle-making protein in three young boys with Duchenne muscular dystrophy, a deadly and irreversible disease, according to results being presented by the drugmaker Sarepta Therapeutics at an investor event this morning.

PAX7 Target Gene Repression is a New Hallmark of Facioscapulohumeral Muscular Dystrophy

نوشته شده توسط musculardystrophynews.com در .

December 22, 2017