Patient's Sudden Death in Pfizer Gene Therapy Study
It is with great sadness we share the information we just received. A patient participating in Pfizer’s Phase 2 DAYLIGHT…
Showing all 30 results
Translarna (Ataluren): Disappointing news for the Duchenne community
Amsterdam, January 26, 2024- The Duchenne community, represented by the World Duchenne Organization, was deeply disappointed to be informed about…
WDO Webinar: Gene Therapy in Duchenne Muscular Dystrophy
This November 7, the World Duchenne Organization (WDO) convened a webinar for its members to explore the evolving landscape of…
FDA Approves Vamorolone as a Treatment for Duchenne Muscular Dystrophy
Today, the U.S. Food and Drug Administration (FDA) has granted its approval for Vamorolone, a new treatment for Duchenne Muscular…
Europe's CHMP Issues Positive Opinion For Santhera’s AGAMREE® (vamorolone) To Treat Duchenne Muscular Dystrophy
The Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion in favor of the approval of…
Translarna (ataluren): Devastating news for the Duchenne community
The Duchenne community, represented by the World Duchenne Organization, is deeply disappointed to be informed about the recommendation of EMA’s…
FDA Approves ELEVIDYS (delandistrogene moxeparvovec-rokl) Gene Therapy for Duchenne Muscular Dystrophy
The FDA granted accelerated approval to ELEVIDYS (delandistrogene moxeparvovec-rokl). This is what you need to know.
Santhera and ReveraGen Complete NDA Submission to FDA and MAA Submission to EMA for Vamorolone
Santhera and ReveraGen Complete NDA Submission to FDA and MAA Submission for EMA for Vamorolone as a potential treatment for DMD.
Pfizer CIFFREO Phase 3 ambulatory trial re-started
The clinical hold on Pfizer CIFFREO Phase 3 ambulatory trial is lifted. They are now working to activate U.S. trial sites.
Edgewise announces results from Phase 1 EDG-5506 study
Edgewise Therapeutics announced positive results from the Phase 1b portion of the first-in-human trial which enrolled individuals with Becker muscular dystrophy.
Topline results of Vamorolone after completing VISION-DMD
Vamorolone given throughout the study showed sustained efficacy across multiple endpoints over 48 weeks and the good safety and tolerability profile was confirmed.
STRIDE data shows Translarna delays loss of ambulation by more than five years in nonsense DMD boys
Data shows Translarna delays loss of ambulation by more than five years in nonsense DMD boys.
Ignite DMD: Data and clinical trial update
Solid Biosciences announced that they have resumed safe dosing at the 2E14 vg/kg dose, and that this patient experienced mild…
FDA approves Sarepta's AMONDYS 45 (casimersen)
Today, Sarepta Therapeutics announced that the U.S. Food and Drug Administration (FDA) has approved AMONDYS 45 (casimersen). AMONDYS 45 is…
Lay summaries of SPITFIRE and THUNDERJET studies available
All results of clinical trials generate new knowledge, and helps to advance the development of treatments. This includes the sharing…
Translarna (ataluren) approved in Russia
PTC Therapeutics today announced that Translarna™ (ataluren) has been granted marketing approval in the Russian Federation for nonsense mutation Duchenne…
Sarepta Announces Clinical Results from MOMENTUM study
Today, Sarepta shared an update for its investigational PPMO SRP-5051 clinical trial study. MOMENTUM, the name of their phase 2…
Genethon gets approval to start gene therapy trial
Genethon is dedicated to design and develop a gene therapy products for rare diseases. On Monday 30th of November it…
Phase 3 PolarisDMD trial with edasalonexent stopped
Our hearts are heavy when the news reached us that the Phase 3 PolarisDMD trial with edasalonexent did not meet…
FDA Orphan Drug Designation for ATL1102 for DMD
Antisense Therapeutics announced that the US Food and Drug Administration (FDA) Office of Orphan Products Development has granted an Orphan…
Santhera stops Phase 3 SIDEROS Study and Development of Puldysa®
Today, Santhera announced the discontinuation of its Phase 3 SIDEROS study with Puldysa® (idebenone) in patients with Duchenne muscular dystrophy…
Santhera tries to get worldwide rights to Vamorolone for all indications – including DMD
Santhera exercises the option to obtain worldwide rights of Vamorolone in Duchenne muscular dystrophy and all other indications. Santhera will…
FDA accepts review of casimersen for DMD patients amenable to skipping exon 45
Today, Sarepta announced the FDA accepted Casimersen for DMD patients amenable to skipping exon 45. The US Food and Drug Administration…
FDA grants accelerated approval for Viltepso (viltolarsen)
Nippon Shinyaku unit NS Pharma today announced the accelerated FDA for Viltepso (viltolarsen). This targeted treatment is created for people…
Webinar: Returning clinical research results to trial participants
VISION-DMD Webinar: Wednesday 19th August 2020 3pm - 4pm CEST Children and their families participating in clinical trials have invested…
Fast Track Designation for Sarepta's Gene Therapy
Sarepta announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin). SRP-9001 is…
CHMP provides Translarna™ (ataluren) Label Update
Translarna is currently indicated for ambulatory Duchenne patients who are over two years of age; the requested extension would have…
Sarepta completes NDA submission for Casimersen
Sarepta today announced they have completed the submission of a rolling New Drug Application (NDA) to the U.S. Food and…
Sarepta announces positive safety and efficacy data from gene therapy trial
Sarepta shared a press release this morning. In here, Sarepta Therapeutics announces positive safety and efficacy data from the SRP-9001…
EC grants Orphan Drug Designation to viltolarsen
Today, Nippon Shinyaku announced that the European Commission (EC) has granted Orphan Drug Designation to viltolarsen. Viltolarsen is an antisense…
