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BioWorld - Friday, January 2, 2026
Home » Topics » Musculoskeletal, BioWorld Science

Musculoskeletal, BioWorld Science
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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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3D cross-section illustration of muscle anatomy
Musculoskeletal

Epirium Bio’s first-in-class 15-PGDH inhibitor restores muscle strength in aged mice

March 19, 2025
Sarcopenia is an age-related condition in which muscle mass and strength decrease, leading to reduced overall physical performance. As fast-twitch muscle fibers are more impacted by age-related decline, strategies aimed at enhancing the regeneration and functionality of these fibers are essential to prevent the progression of sarcopenia.
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Illustration of man holding magnifying glass to human body model showing muscle anatomy
Neurology/psychiatric

New in vitro model of inclusion body myositis based on human iPSC-myotubes

March 17, 2025
Inclusion body myositis (IBM) is an inflammatory myopathy causing proximal and distal muscle weakness. IBM’s cause remains unknown, lacking validated models, biomarkers and effective treatment strategies. Histopathological studies identified inflammatory infiltrates, rimmed vacuoles, and mitochondrial changes in the muscles of IBM patients.
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Justin Rubio working in the lab
Inflammatory

Inflammation appears to cause mutations linked to MS progression

March 10, 2025
By Tamra Sami
For the first time, researchers have identified that inflammation – long associated with multiple sclerosis (MS) – appears to cause increased mutations that damage neurons linked to MS progression. Researchers at the Florey Institute and the University of Melbourne studied MS brain lesions, which are areas of past or ongoing brain inflammation that are visible as spots on MRI scans.
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