Gene Therapy for Juvenile Batten Disease Gets Orphan Drug Designation – Rare Disease Report
Rare Disease Report |
Gene Therapy for Juvenile Batten Disease Gets Orphan Drug Designation
Rare Disease Report This is the 4th gene therapy the company has in development that has obtained an Orphan Drug Designation. The other 3 are being developed to treat recessive dystrophic epidermolysis bullosa and both sanfilippo syndrome type A and type B. The company … |
