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BioWorld - Tuesday, February 24, 2026
Home » Topics » Musculoskeletal, BioWorld Science

Musculoskeletal, BioWorld Science
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Zebrafish embryo image produced using a hyperspectral multipoint confocal fluorescence microscope.
Neurology/psychiatric

Dock7-mutant zebrafish show disrupted skeletal muscle myofiber structure and reduced locomotion

March 28, 2025
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.
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Illustration of DNA double helix and motorized wheel chair
Neurology/psychiatric

CLS-189, a potential best-in-class HPGDS inhibitor for the treatment of DMD

March 28, 2025
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).
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Neurology/psychiatric

PBGENE-DMD restores dystrophin functioning in DMD

March 27, 2025
Precision Biosciences Inc. recently presented a new gene-editing approach, PBGENE-DMD, which could allow life-long benefits to patients with Duchenne muscular dystrophy (DMD).
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Illustration of muscle tissue anatomy
Musculoskeletal

EDG-4131 active in model of Becker muscular dystrophy

March 26, 2025
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.
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3D dollar sign
Neurology/psychiatric

Augustine Therapeutics closes $85M series A for HDAC6 inhibitors

March 26, 2025
By Nuala Moran
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.
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3d illustration of human body muscle tissue anatomy
Neurology/psychiatric

Irodanoprost improves muscle function and histology in experimental DMD

March 25, 2025
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.
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Neurology/psychiatric

New canine model of Duchenne muscular dystrophy reported

March 24, 2025
Researchers from Suzhou Genassist Therapeutics Co. Ltd. have developed a novel canine model of Duchenne muscular dystrophy (DMD), expected to be more predictive of disease pathogenesis and treatment efficacy.
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Genetic/congenital

CMT2E murine model reveals early axonal damage

March 21, 2025
Charcot-Marie-Tooth disease type 2E (CMT2E) is a slow and progressive neuropathy characterized by axonal dysfunction. Its clinical phenotype includes muscle weakness and atrophy, sensory loss and reduced nerve conduction velocity, among others.
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Muscular dystrophy
Neurology/psychiatric

Insmed reports preclinical data on INS-1201 for DMD

March 20, 2025
Investigators from Insmed Inc. have presented new preclinical data on the efficacy of their adenoviral vector (AAV9)-based gene therapy INS-1201 for the treatment of Duchenne muscular dystrophy (DMD).
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Neurology/psychiatric

DMD base editing candidate shows safety and efficacy in preclinical models

March 20, 2025
At this week’s Muscular Dystrophy Association Clinical and Scientific Conference in Dallas, researchers from Suzhou Genassist Therapeutics Co. Ltd. presented preclinical data for GEN-6050X (ss.AAV9.oTAM and ss.AAV9.hE50-sgRNA).
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