Previous studies have shown that protein expression of DOCK7 is increased in skeletal muscle biopsies from patients with Duchenne muscular dystrophy (DMD), leading researchers from the University of Alabama at Birmingham and affiliated organizations to assess the functional impact of DOCK7 on normal muscle and embryonic development of zebrafish.

Researchers from the University of Queensland recently provided details on the discovery and preclinical characterization of a new hematopoietic prostaglandin D2 synthase (HPGDS) inhibitor, CLS-189, being developed for the treatment of Duchenne muscular dystrophy (DMD).

Wave Life Sciences Ltd. plans to file with the U.S. FDA for accelerated approval of WVE-N531, an exon skipping oligonucleotide for boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping, a decision based on favorable data from the phase II Forward-53 study. The analysis was conducted after 48 weeks of treatment with 10 mg/kg of the drug dosed every two weeks. Forward-53 achieved all trial goals, turning up sustained exon skipping, muscle concentrations, and dystrophin restoration through 48 weeks and a 61-day tissue half-life that supports giving the DMD therapy once a month.

Researchers from Edgewise Therapeutics Inc. have previously developed and characterized a novel bmx mouse model of Becker muscular dystrophy (BMD), which harbors a deletion of murine Dystrophin exons 45-47 on a C57/BL6J background.

Eight months after announcing the $18.5 million first tranche of its series A, Augustine Therapeutics has closed the oversubscribed round at $85 million and is now ready to begin clinical development of its novel histone deacetylase-6 (HDAC6) inhibitors.

Nature Cell Co. Ltd. won U.S. FDA breakthrough therapy designation of its autologous adipose-derived mesenchymal stem cell therapy, Jointstem, March 20, becoming the first Korean company to earn the label in the field of cell therapy.

Prostaglandins induce the regeneration of muscle in rodents and humans through the prostaglandin E2 receptor EP4 subtype receptor, but this therapeutic pathway's potential is limited due to systemic tolerability. Researchers from Mesentech Inc. recently presented new results on their prostaglandin E2 receptor EP4 subtype receptor agonist irodanoprost trying to address this limitation issue.

Eight months after announcing the $18.5 million first tranche of its series A, Augustine Therapeutics has closed the oversubscribed round at $85 million and is now ready to begin clinical development of its novel histone deacetylase-6 (HDAC6) inhibitors.