Capricor Therapeutics Submits Application for Treatment of DMD Cardiomyopathy
Capricor Therapeutics, a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, announced today that it has initiated its...
Satellos Produced Near-Normal Levels of Dystrophin in Experiment with Dogs with DMD with SAT-3247
According to the announcement made by Satellos Bioscience Inc., in an experiment conducted on dogs with DMD, it was seen that dystrophin was produced...
CRISPR-Cas13 Could Accelerate Studies on RNA-Targeted Therapies
In recent years, the scientific community has made significant strides in the field of gene editing, particularly through the development of the CRISPR (Clustered...
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...
Cures of Duchenne (List of All Researches)
Dystrophin Gene Therapy
MechanismCompanyDrugStatus
Micro-dystrophin Gene Replacement Sarepta TherapeuticsElevidys/SRP-9001ApprovedMicro-dystrophin Gene ReplacementPfizerPF-06939926DiscontinuedMicro-dystrophin Gene ReplacementGenethon (Sarepta)GNT-0004ClinicalMicro-dystrophin Gene ReplacementRegenxBioRGX-202ClinicalMicro-dystrophin Gene ReplacementSolid BiosciencesSGT-001ClinicalMicro-dystrophin Gene ReplacementSolid BiosciencesSGT-003ClinicalMicro-dystrophin Gene ReplacementUltragenyxUX810PreclinicalMicro-dystrophin Gene ReplacementInsmedINS1201PreclinicalMicro-dystrophin Gene...