March 2, 2021
Sarepta Therapeutics has provided information on the Essence clinical trial currently recruiting in Canada for boys with deletions amenable to exon 45 and exon 53 skipping. The purpose of this Phase III research study is to evaluate the safety and effectiveness of SRP-4045 and SRP- 4053, Sarepta’s exon 45- and exon 53-skipping investigational drugs. Please […]
February 25, 2021
Italfarmaco delivered a presentation at the International Conference on Duchenne and Becker Muscular Dystrophy where their Chief Medical Officer, Dr. Bettica, presented the long term data following patients who have received givinostat in addition to steroid treatment for seven years: “We are very encouraged to see that the long-term study with Givinostat continues to show […]
February 25, 2021
Sarepta Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has approved the Amondys 45 (casimersen) for patients amenable to skipping exon 45. This extends their commercial reach to approximately 30% of all Duchenne muscular dystrophy patients. “This is an important day for Sarepta and, far more importantly, for the patients that we […]
February 23, 2021
Italfarmaco has shared a letter to the Duchenne community which provides an update about navigating trial visits that cannot be adapted for the home setting: “All of us at Italfarmaco would like to thank the families participating in the Phase 3 EPIDYS trial and in the open-label extension study of givinostat in Duchenne muscular dystrophy […]
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 [email protected] 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. […]
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 […]
