Long-Term Follow Up of Patients Receiving Novel Gene Therapy for SMA Type I
Researchers recently published the long-term outcomes of patients who received the investigational drug AVXS-101 – an adeno-associated virus serotype 9 mediated gene replacement therapy.
Preventing the Development of Muscular Dystrophy Through Surrogate Gene Therapy
Researchers identify a key regulatory protein implicated in Galgt2 overexpression and begin to elucidate its protective mechanism against muscular dystrophy.
