Interim results from a small group of children in a Phase I/II trial are essentially in line with that of Elevidys, according ...

Gene therapies, exon-skipping drugs and next-gen steroids are reshaping Duchenne muscular dystrophy care, targeting broader ages and mutations. We are witnessing the fastest pivot in rare-disease ...

With FDA platform technology designation for its viral vector, SRPT can fast-track other therapies using the same ...

RegenXBio has shared updated phase 1/2 data on its Duchenne muscular dystrophy (DMD) gene therapy, providing evidence that ...

The Platform Technology Designation, which precedes the current FDA leadership, is designed to streamline the drug ...

The former workers cited various reasons why they think Novo was cautious: its conservative culture and history of investing ...

Abeona's ZEVASKYN approval and a $3.1 million price point are undervalued by the market. Find out why ABEO stock is a Buy.