DMD News Briefs

MDA officials

Possible Approval of First DMD Gene Therapy Creates Buzz at MDA Conference

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…

gene therapy concept

Gene Transfer Shows Promising Results for DMD

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…

Wheelchair

Novel Drug for DMD Will Undergo an Advisory Committee Review

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…

DMD history

Clinical Trial Assessing Novel Antisense Oligonucleotide for DMD Underway

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…

Case Report

DMD-Associated Myocardial Injury Treated With Steroids

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…

DMD history

CAP-1002 Shows Disease Modification in Patients With Later-Stage DMD

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…

bicycle exercise

Aerobic Cycle Training May Improve Endurance in Boys With DMD

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…

FDA Headquarters

FDA Grants Orphan Drug Designation for Potential New DMD Therapeutic

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…

DMD history

New Model May Explain Variability in Duchenne Muscular Dystrophy Progression

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…

biomarker

Novel Marker for DMD May Be Superior to CK-M

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…

Next post in DMD News Briefs