Wave Life Sciences announced today positive data following 48 weeks of dosing in the FORWARD-53 clinical trial evaluating the investigational molecule …
Wave Life Science Announce Positive Data from FORWARD-53 Clinical TrialRead More
This is the latest news about the development and use of exon skipping in Duchenne muscular dystrophy. There’s also lots of information and videos explaining these potential treatments in our information pages.
March 26, 2025 by John Marrin
Wave Life Sciences announced today positive data following 48 weeks of dosing in the FORWARD-53 clinical trial evaluating the investigational molecule …
Wave Life Science Announce Positive Data from FORWARD-53 Clinical TrialRead More
March 26, 2025 by John Marrin
Dyne Therapeutics recently announced new long-term clinical data from its ongoing Phase 1/2 DELIVER trial of DYNE-251 demonstrating sustained …
Dyne Therapeutics Announce New Long Terms Clinical Data from DELIVER Trial of DYNE 251Read More
March 24, 2025 by Mehreen Arif
Entrada Therapeutics Receives Authorization in the United Kingdom to Initiate ELEVATE-45-201, a Phase 1/2 Multiple Ascending Dose Clinical Study of …
March 12, 2025 by John Marrin
PepGen's has released an update regarding the CONNECT clinical trials for PGN-EDO51 in Duchenne muscular dystrophy (DMD). PepGen has announced a …
PepGen Announces Update to Phase 2 CONNECT2-EDO51 Study in Patients with DMDRead More
November 7, 2024 by John Marrin
We are disappointed to learn of the news regarding Sarepta’s SRP-5051 MOMENTUM study. Sarepta has decided to stop developing SRP-5051, a potential …
Sarepta Announces the Discontinuation of MOMENTUM Study and Development of SRP-5051Read More
October 11, 2024 by John Marrin
Wave Life Sciences has shared their promising new interim results for the ongoing FORWARD-53 trial. The ongoing open-label trial, involves eleven boys …
August 5, 2024 by John Marrin
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 …
June 28, 2024 by John Marrin
Entrada Therapeutics, a company developing treatments for rare diseases, reports encouraging preliminary data from a Phase 1 clinical trial …
March 5, 2024 by John Marrin
PepGen, a biotech company developing innovative oligonucleotide therapies for severe neuromuscular and neurological diseases, has received approval …
PepGen Receives UK CTA Clearance from MHRA to Begin CONNECT2-EDO51 Phase 2 Clinical TrialRead More
January 31, 2024 by John Marrin
On the Monday the 29th of January, Sarepta Therapeutics announced positive data from Part B of the MOMENTUM study. The MOMENTUM Study is a global …
January 11, 2024 by John Marrin
Dyne Therapeutics have reported their initial clinical data from the DELIVER trial of DYNE-251 in patients with Duchenne muscular dystrophy who are …
January 11, 2024 by John Marrin
PepGen Inc., a clinical-stage biotechnology company advancing the next-generation of oligonucleotide therapies, today announced that the first patient …
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