Owing to a serious adverse event in a patient, the U.S. Food and Drug Administration (FDA) has put another clinical hold on a Phase 1/2 clinical trial of SGT-001, an investigational gene therapy for Duchenne muscular dystrophy (DMD). Solid Biosciences, the company developing SGT-001, is working…
A novel one-time gene therapy that leads to the production of a dystrophin-related protein called utrophin prevents muscle deterioration and results in muscle regeneration in animal models of Duchenne muscular dystrophy (DMD) without triggering an immune response, a new study reports. The study, “Non-immunogenic…
Increasing the amount of a protein called sarcospan may help to protect against heart failure in children with Duchenne muscular dystrophy (DMD), a study in mice suggests. The study, “Stabilization of the cardiac sarcolemma by sarcospan rescues DMD-associated cardiomyopathy,” was published in the journal JCI Insight. Despite significant advances…
Catabasis Pharmaceuticals announced new preclinical data showing that, in a mouse model of Duchenne muscular dystrophy (DMD), the small molecule edasalonexent preserved bone health, whereas the corticosteroid prednisolone caused bone loss. An ongoing double-blind and randomized Phase 3 trial (NCT03703882), called PolarisDMD, is evaluating…
Biotechnology company Vertex has acquired Exonics Therapeutics, which focuses on using gene-editing technologies to treat Duchenne muscular dystrophy (DMD) — a move that is being hailed as a positive step toward getting these therapies into clinics. Exonics was co-founded in 2017 by CureDuchenne, a nonprofit organization…
People with facioscapulohumeral muscular dystrophy (FSHD) are “very enthusiastic” about participating in clinical trials and studies, according to researchers, who report that foot and leg, and shoulder and arm weakness have the greatest impact on patients’ quality of life. The investigators said their study found 93% of patients were…
Very young boys with Duchenne muscular dystrophy (DMD) have measurable differences in motor skills, which could have implications for the early detection of DMD and for evaluating how treatments affect their development as opposed to the natural course of the disease, a study shows. Findings of the study, “…
RG6206 (BMS-986089), a blocker of muscle growth-inhibitor myostatin, is safe to use in boys with Duchenne muscular dystrophy (DMD), results from a Phase 1b/2 trial show. A Phase 2/3 trial of the drug (NCT03039686) is actively recruiting patients. Moreover, the results showed that the compound was able to reduce…
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