Capricor Therapeutics Announces Positive 4-Year Data of Deramiocel (CAP-1002) in Duchenne Muscular Dystrophy
Capricor Therapeutics, a biotechnology company developing transformative cell and exosome-based therapeutics for rare diseases, today announced positive four-year safety and efficacy results from its...
Capricor Therapeutics Announces BMD Orphan Drug Designation and DMD Program Regulatory Development for Deramiocel (CAP-1002)
Capricor Therapeutics, a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that the U.S. Food and...
Sarepta Reported Elevidys Gene Therapy for Duchenne Muscular Dystrophy will not be used in non-ambulatory patients
ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy, has received a safety update from Sarepta Therapeutics, the leader...
New Positive Functional Data From The RGX-202 Affinity Duchenne® Trial Phase 1/2 Reported By Regenxbio
The Regenxbio press statement stated that the findings, which came from five patients in the AFFINITY DUCHENNE trial who were between the ages of...
PepGen Announces It Will Halt Clinical Trials of Exon 51 Skipping Study
PepGen, a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies, today announced that based on the levels of dystrophin protein measured in...