REGENXBIO, a leading clinical-stage biotechnology company focused on advancing transformative gene therapies, announced in late November that the …
REGENXBIO Announces Pivotal Phase of AFFINITY DUCHENNE Clinical Trial for RGX-202 Read More
This is the latest news about gene therapy development for Duchenne muscular dystrophy. There’s also lots of information and videos explaining these potential treatments in our information pages.
Clinical Trial Update – PepGen Announces Positive Data from Ongoing CONNECT1-EDO51 Phase 2 Trial for Treatment of Duchenne Muscular Dystrophy
PepGen Inc., announced on July 30th 2024, positive clinical data from the first dose cohort (5 mg/kg) of PGN-EDO51, its lead investigational candidate …
Clinical Trial Update – RegenxBio Announces New Positive Data From Affinity Duchenne Trial of RGX-202
On the 1st of August, RiogenxBio provided a positive update on the interim safety and efficacy data collected from their Phase I/II AFFINITY DUCHENNE …
Important Update on Pfizer’s CIFFREO Phase 3 Clinical Trial for DMD Gene Therapy
In June, Pifzer announced that their CIFFREO Phase 3 clinical trial for Duchenne muscular dystrophy (DMD) gene therapy did not meet its primary and …
Important Update on Pfizer’s CIFFREO Phase 3 Clinical Trial for DMD Gene TherapyRead More
Sarepta Therapeutics Announces Positive Update on ELEVIDYS (delandistrogene moxeparvovec-rokl) Regulatory Progress for Duchenne Muscular Dystrophy (DMD)
Sarepta Therapeutics, Inc. today provided an update on the regulatory progress of ELEVIDYS (delandistrogene moxeparvovec-rokl), its gene therapy for …
Sarepta Therapeutics Announces U.S. FDA Acceptance of an Efficacy Supplement to Expand the ELEVIDYS Indication
Sarepta Therapeutics, Inc, today announced the U.S. Food and Drug Administration (FDA) has accepted and filed the company's ‘Efficacy Supplement’ to …
Solid Biosciences Receives FDA Clearance for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003
Solid Biosciences Receives FDA Clearance for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003Solid Biosciences Inc., a leading life sciences …
Roche UK issues a statement following Sarepta’s Press release regarding ELEVIDYS FDA decision
Roche UK issues a statement following Sarepta's Press release regarding ELEVIDYS FDA decisionFollowing the press release from Sarepta released on 22nd …
Sarepta Therapeutics Announces FDA Approval of ELEVIDYS, the First Gene Therapy to Treat Duchenne Muscular Dystrophy
Sarepta Therapeutics announced U.S. Food and Drug Administration (FDA) accelerated approval of ELEVIDYS (delandistrogene …
Roche UK issues statement to the Duchenne Community following Sarepta’s Update on Regulatory Review of SRP-9001 gene therapy.
Roche UK issues statement to the Duchenne Community following Sarepta's Update on Regulatory Review of SRP-9001 gene therapy. Following the update …
Sarepta Therapeutics Announces Update on Regulatory Review of SRP-9001
Sarepta Therapeutics Announces Update on Regulatory Review of SRP-9001Sarepta Therapeutics, today provided the following update on the Biologics …
Sarepta Therapeutics Announces Update on Regulatory Review of SRP-9001Read More
Sarepta Therapeutics Announces Positive Vote from U.S. FDA Advisory Committee Meeting for SRP-9001 Gene Therapy to Treat Duchenne Muscular Dystrophy
Sarepta Therapeutics Announces Positive Vote from U.S. FDA Advisory Committee Meeting for SRP-9001 Gene Therapy to Treat Duchenne Muscular …
