DALLAS, Texas—Despite a late-stage regulatory hiccup, pharmaceutical executives and patient advocates at a neuromuscular disease meeting here predict the US Food and Drug Administration (FDA) will still approve the first-ever gene therapy for Duchenne muscular dystrophy (DMD) as expected on May 29, 2023. SRP-9001 (delandistrogene moxeparvovec), developed by Sarepta Therapeutics, was easily the chief topic…
A novel gene transfer therapy could yield important benefits with a tolerable safety performance for treating patients diagnosed with Duchenne muscular dystrophy (DMD), according to a study recently published as a poster in the MDA Clinical & Scientific Conference of 2023. “Findings from Study 102 reinforce that delandistrogene moxeparvovec has a favorable benefit–risk profile, with…
A recently established committee will discuss a novel therapeutic option for Duchenne muscular dystrophy (DMD), according to a press release made by Sarepta Therapeutics, Inc. “It is our understanding that as one of the first gene therapy BLAs founded on a surrogate endpoint, the advisory committee will primarily relate to the totality of evidence supporting…
A new clinical trial, AVANCE1, is recruiting adult and pediatric patients with Duchenne muscular dystrophy (DMD) to investigate the safety, pharmacokinetics (PK), and pharmacodynamics of a novel antisense oligonucleotide called SQY51. The trial, sponsored by SQY Therapeutics and Biotrial, is recruiting up to 12 participants to initially receive escalating intravenous infusions of SQY51 every 2…
Early treatment with corticosteroids quickly reversed ST-segment elevation and troponin levels in a 9-year-old male with Duchenne muscular dystrophy (DMD)-associated myocardial injury, according to a recently published case report in the Journal of Cardiothoracic Surgery. DMD cardiomyopathy, characterized by muscle inflammation and the deposition of fibrotic tissue, is currently the leading cause of death among…
The investigational Duchenne muscular dystrophy (DMD) therapy, CAP-1002, continues to be beneficial and well tolerated in the long term, according to results from the open-label extension study of the phase 2 HOPE-2 trial (HOPE-2-OLE). These results will be presented at the 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference taking place from March 19…
It may be possible to counter muscle disuse and improve endurance in boys with Duchenne muscular dystrophy (DMD) using aerobic cycle training, according to new results that will be presented at the 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference. The results are based on a home-based, cycle-training program developed specifically for boys with…
The US Food and Drug Administration (FDA) granted orphan drug designation for a new induced pluripotent stem cell therapeutics for Duchenne muscular dystrophy (DMD). The therapeutic called GIVI-MPC could create new skeletal muscles expressing full-length dystrophin protein, the missing protein in DMD. Read more about the etiology of DMD Rauf Ashraf, the chief executive officer…
Researchers from the United States and Canada developed a new model that could explain the variability in the rate of disease progression and response to treatment in patients with Duchenne muscular dystrophy (DMD). The new model could help define better inclusion criteria in clinical trials, stratify patients for subgroup analysis, and monitor disease progression in…
A molecule detectable in urine could become a novel biomarker for Duchenne muscular dystrophy (DMD), according to a study recently published in Neuromuscular Disorders. “Here, we demonstrated that the elevated titin in urine is directly associated with the lack of dystrophin and urine titin responses to drug treatment,” the authors wrote. This experimental study included…
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