REGENXBIO announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial. Updates include positive functional, safety and biomarker …
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.
Action Duchenne’s Statement Regarding Sarepta’s Sad Announcement
We are deeply saddened to learn of the passing of a young man who received ELEVIDYS gene therapy for Duchenne muscular dystrophy. Our hearts go out to …
Action Duchenne’s Statement Regarding Sarepta’s Sad AnnouncementRead More
Roche Shares Top-line Results from Year Two of the EMBARK Phase 3 Clinical Trial.
Roche have provided an update on the latest top-line results from the second year of their EMBARK Phase 3 clinical trial (NCT05096221) investigating …
Roche Shares Top-line Results from Year Two of the EMBARK Phase 3 Clinical Trial.Read More
Solid Biosciences Shares 90 Day Update From SGT-003 Gene Therapy Trial
We're excited to share news that Solid Biosciences has released initial data from its Phase 1/2 INSPIRE DUCHENNE clinical trial, assessing the safety …
Solid Biosciences Shares 90 Day Update From SGT-003 Gene Therapy TrialRead More
REGENXBIO Announces Pivotal Phase of AFFINITY DUCHENNE Clinical Trial for RGX-202
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
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-003 Solid Biosciences Inc., a leading life …
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 decision Following the press release from Sarepta released on …
