Hearing a parent agonize over the plight of their child who suffers from a rare disease is heart wrenching. For these families, research into rare disease drugs hasn’t come close to “reaching an extreme”.
Category: NASDAQ:SRPT
A Gene Therapy Appears ToReplaceMissing ProteinIn Muscular Dystrophy Patients
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 Sare…
