New Gene Therapy Study at Indiana University Offers Hope for Duchenne Muscular Dystrophy
Dr. Renzhi Han, a professor of pediatrics at Indiana University, is working on a new gene therapy for the treatment of Duchenne (DMD) and...
Cure of Duchenne (Research Overview)
Dystrophin Gene Therapy
Mechanism
Company
Drug
Status
Micro-dystrophin Gene Replacement
Sarepta Therapeutics
Elevidys/SRP-9001
Approved
Micro-dystrophin Gene Replacement
Pfizer
PF-06939926
Discontinued
Micro-dystrophin Gene Replacement
Genethon (Sarepta)
GNT-0004
Clinical
Micro-dystrophin Gene Replacement
RegenxBio
RGX-202
Clinical
Micro-dystrophin Gene Replacement
Solid Biosciences
SGT-001
Clinical
Micro-dystrophin Gene Replacement
Solid Biosciences
SGT-003
Clinical
Micro-dystrophin Gene Replacement
Ultragenyx
UX810
Preclinical
Micro-dystrophin Gene Replacement
Insmed
INS1201
Preclinical
Dystrophin Exon...