February 5, 2021
PTC Therapeutics has shared the results of the dystrophin study (Study 045), which assessed dystrophin levels in patients with nonsense mutation Duchenne muscular dystrophy treated with Translarna™ (ataluren). Click here to see the full press release. The Duchenne community is invited to hear more about the study results on their conference call: When: Thursday, February […]
February 3, 2021
Santhera Pharmaceuticals has shared an update with the Duchenne community regarding the Phase 2b trial for vamorolone, which the company expects to have data from in the second quarter of 2021. Read the full press release here. Are you participating in one of the vamorolone clinical trials? Contact Suzanne Gaglianone at Suzanne.gaglianone@reveragen.com to opt-in to […]
January 11, 2021
On January 7, Sarepta Therapeutics announced their topline results from their gene therapy candidate SRP-9001. This study is an ongoing, randomized, double-blinded, placebo-controlled trial evaluating the safety, efficacy and tolerability of a single dose of SRP-9001 in 41 Duchenne patients aged 4-7. The study met its biological primary endpoint of micro-dystrophin expression at 12 weeks. […]
January 11, 2021
January 4, 2021
December 7, 2020
Sarepta has announced encouraging interim results with their SRP-5051 exon skipping molecule for both ambulatory (walking) and non-ambulatory (non-walking) Duchenne patients amenable to exon 51 skipping ages 4-21 years. This new second-generation exon skipping molecule in early clinical trials shows higher tissue exposure than Eteplirsen, with a favourable safety profile. They continue to test higher […]
November 20, 2020
There’s good news for the Duchenne community as Lilly and Precision Biosciences announced a large investment in a genome editing platform of which Duchenne is one of the genetic disorders they will focus on. This research collaboration using ARCUS genome editing technology will initially include three gene targets, with right to select three additional gene […]
November 17, 2020
ReveraGen BioPharma, the developer of vamorolone, received a 3.3 million dollar grant from the National Institutes of Health (NIH) to support the commercialization of this safer alternative for corticosteroids treatment. This will advance vamorolone towards FDA submission and commercialization in the USA. This is excellent news for the Duchenne community and is still pending their […]
November 9, 2020
About SRP-9001 (AAVrh74.MHCK7.micro-dystrophin) SRP-9001 is an investigational gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. Sarepta is responsible for global development and manufacturing of SRP-9001 and plans to commercialize SRP-9001 in the United States. In December 2019, the Company announced a licensing agreement […]
October 27, 2020
Catabasis has announced its Phase 3 PolarisDMD trial discontinuation with edasalonexent, an anti-inflammatory medication under investigation for Duchenne muscular dystrophy. The stop to the PolarisDMD trial was due to a lack of efficacy (no benefit compared to placebo) and not any safety issues. Catabasis is extremely disappointed by this outcome and thanks to the Duchenne […]
