The U.S. Food and Drug Administration (FDA) has granted both fast track and orphan drug designations to Scholar Rock‘s apitegromab as a potential treatment for facioscapulohumeral muscular dystrophy (FSHD), a disease type that characteristically affects muscles of the face and upper body. The experimental…
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September is Muscular Dystrophy Awareness Month, and advocates are launching campaigns to educate people about the different types of rare genetic muscle disorders that comprise muscular dystrophy (MD) — and to fundraise to fuel research. Still, the focus is on spotlighting those living with MD, Kelly Berger, community engagement…
For the first time, a person with Duchenne muscular dystrophy (DMD) has been given an experimental gene-editing therapy, called PBGENE-DMD, that’s designed to treat the neurodegenerative disease by altering the patient’s own genetic code. Precision Biosciences, the U.S. company developing PBGENE-DMD, announced that dosing had begun in the…
The U.S. Food and Drug Administration (FDA) is extending its review of deramiocel, an experimental cell therapy for Duchenne muscular dystrophy (DMD) as it reviews additional data. Capricor Therapeutics, the company developing deramiocel, said it amended its application seeking FDA approval to include new long-term data from…
Epicrispr Biotechnologies has raised $90 million in financing to support late-stage clinical testing of EPI-321, the company’s epigenetic treatment candidate for facioscapulohumeral muscular dystrophy (FSHD). “This financing marks a pivotal milestone for Epicrispr as we advance EPI-321 and the next generation of programmable epigenetic medicines,” Amber Salzman, PhD,…
The U.S. Food and Drug Administration (FDA) has allowed the enrollment of up to 30 additional boys with Duchenne muscular dystrophy (DMD) in a clinical study testing an experimental stem cell therapy. The Phase 2 trial (NCT06579352) testing the treatment from Signature Biologics has enrolled five boys,…
A U.S. biopharmaceutical company is gearing up to launch its first clinical trial of a novel cell therapy designed to restore lost muscle cells in people with Duchenne muscular dystrophy (DMD). Texas-based IPS Heart announced that it is preparing an investigational new drug application or IND — a formal…
When muscle fibers fail to properly seal tiny tears in their membrane, as occurs in some forms of muscular dystrophy, the resulting leakage can change the surrounding environment and attract immune cells that contribute to further muscle damage, a mouse study suggests. The findings suggest that the tissue surrounding…
The University of Alabama at Birmingham (UAB) has launched an expanded access program to make the experimental exon-skipping therapy delpacibart zotadirsen (del-zota) available to qualifying patients with Duchenne muscular dystrophy (DMD). Del-zota, which is being developed by Avidity Biosciences, is specifically designed to treat DMD in people…
An advisory committee to the U.S. Food and Drug Administration (FDA) has voted that available evidence does not support the effectiveness of the experimental cell therapy deramiocel for treating heart muscle disease in people with Duchenne muscular dystrophy (DMD). In a company press release, Capricor Therapeutics,…
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