Asklepios Biopharmaceutical (Askbio) has dosed the first participant in the second group of a Phase 1/2 trial testing AB-1003, an experimental gene therapy for limb-girdle muscular dystrophy (LGMD) type 2I/R9. The Phase 1/2 LION-CS101 trial (NCT05230459) is assessing AB-1003’s safety and tolerability in adults with LGMD2I/R9. Enrollment…
The U.S. Food and Drug Administration (FDA) has accepted Capricor Therapeutics’ application seeking approval of deramiocel — a cell therapy developed by the biotech company to treat heart muscle disease in people with Duchenne muscular dystrophy (DMD) — and granted it priority review that will speed the agency’s…
Adolescents and young adults with Duchenne (DMD) or Becker muscular dystrophy (BMD) who have more siblings typically report being better prepared to transition to adult life, a new study has found. Findings show people with DMD or BMD had the most difficulty with activities of daily living, education,…
Once a week treatment with a corticosteroid was seen to lower some biomarkers of muscle damage and inflammation, helping to improve muscle function in people with some forms of muscular dystrophy, according to a pilot study in the U.S. Prednisone was given once weekly for six months to 19…
Note: This story was updated Jan. 17, 2025, to clarify the company’s comments on gene therapies, which generally deliver a smaller form of dystrophin. The U.S. Food and Drug Administration (FDA) has granted IPS Heart’s GIVI-MPC — a stem cell therapy designed to make new muscle tissue in people…
During 2024, Muscular Dystrohy News Today covered the latest scientific studies, advances in treatment, and clinical trials for the multiple types of muscular dystrophy (MD). Here is a list of the top 10 most-read stories we published last year. No. 10 – Patient death prompted pause in DMD…
A new technology called StitchR effectively restored the production of large muscle proteins dystrophin and dysferlin in mouse models of Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophy (LGMD), according to a study. The results have potential implications for gene therapies. The technology works by delivering two halves…
Despite positive trial data, Sarepta Therapeutics has decided to stop the clinical development of SRP-5051 (vesleteplirsen), an exon 51-skipping therapy for some people with Duchenne muscular dystrophy (DMD). While increases in dystrophin levels seen with the exon-skipping treatment were encouraging, according to the company, concerns over the…
MDL-101, Modalis Therapeutics’ epigenetic editing therapy for LAMA2-related congenital muscular dystrophy (LAMA2-CMD), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). The designation is intended to encourage the development of therapies for serious or life-threatening rare diseases, which are those affecting fewer than…
Capricor Therapeutics has started its rolling submission seeking U.S. approval of deramiocel, a cell therapy candidate to treat cardiomyopathy, a disease of the heart muscle, in people with Duchenne muscular dystrophy (DMD), the company announced. Rolling submission means that Capricor will be submitting parts of the process to…
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