March 16, 2021
Solid Biosciences has provided an update on the safety and efficacy data from their IGNITE DMD Phase I/II clinical trial: We are pleased to share an update regarding the safety and efficacy data from our ongoing IGNITE DMD phase I/II clinical trial, as announced in the press release issued this afternoon. Additionally, we are excited to announce […]
March 16, 2021
March 15, 2021
Recent studies on Duchenne muscular dystrophy (DMD) have greatly deepened our understanding of the primary and secondary pathogenetic mechanisms. Guidelines for the multidisciplinary care for Duchenne that address obtaining a genetic diagnosis and managing the various aspects of the disease have been established. In addition, a number of therapies that aim to restore the missing […]
March 12, 2021
The Hospital for Sick Children (SickKids) in Toronto is currently recruiting boys with Duchenne muscular dystrophy aged 4-7 with mutations amenable to exon 53 skipping for the NS Pharma, Inc. RACER53 clinical trial investigating the new drug, viltolarsen. Click here to learn more and find out if you might qualify for this study: racer53dmdstudy.com. RACER53 Study Overview […]
March 10, 2021
March 3, 2021
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 […]
