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BioWorld - Monday, February 16, 2026
Home » Topics » Disease categories and therapies » Musculoskeletal

Musculoskeletal
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Shares slide as Avidity identifies ‘extremely rare’ SAE in DM1 trial

March 30, 2023
Shares of Avidity Biosciences Inc. (NASDAQ:RNA) dropped 15% March 30 as the company disclosed details of the serious adverse event (SAE) that prompted a partial clinical hold in September 2023 on the phase I/II trial testing AOC-1001 in adults with myotonic dystrophy type 1 (DM1).
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Light micrograph of skeletal muscle.
Musculoskeletal

MYTHO identified as novel FoxO-dependent gene and regulator of skeletal muscle autophagy

March 29, 2023
Autophagy plays a critical role in the regulation of skeletal muscle integrity; however, the molecular mechanisms regulating autophagy are not fully understood. In a recently published study, researchers from McGill University Health Centre and affiliated organizations aimed to investigate these mechanisms and identify novel regulators of autophagy and skeletal muscle integrity.
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Neurology/Psychiatric

New murine DMD model resembles the severe phenotype seen in humans

March 29, 2023
The main features of Duchenne muscular dystrophy (DMD) are muscle weakness and...
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Musculoskeletal

Study probes intersection of immune and muscle aging

March 27, 2023
By Anette Breindl
Restoring levels of the immune modulator mesencephalic astrocyte-derived neurotrophic factor (MANF) in the muscles of aged animals improved their regenerative capacity by promoting repair-associated injury responses, researchers from the University of Lisbon have discovered.
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Illustration of leg anatomy with target around knee
Inflammatory

Partially blocking inflammatory receptor could lead to treatment for osteoarthritis

March 24, 2023
By Helen Albert
Partially blocking a receptor that helps regulate the activity of the inflammatory cytokine molecule interleukin-6 (IL-6) seems to promote tissue regeneration and block degeneration in a model of osteoarthritis. As reported in the March 22, 2023, issue of Science Translational Medicine, the receptor, called glycoprotein 130 (gp130), regulates both positive and negative inflammatory responses that can help regenerate tissue, but also cause degeneration.
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Person sitting in wheelchair

Almost, but not quite: Adcom is mixed on Biogen’s ALS drug

March 23, 2023
By Lee Landenberger
Tofersen’s development is progressing in fits and starts. That was evident at the U.S. FDA’s March 22 meeting of the Peripheral and Central Nervous System Drugs Advisory Committee, which unanimously agreed Biogen Inc.’s failed phase III study predicted a clinical benefit in treating amyotophic lateral sclerosis (ALS) that includes the rare superoxide dismutase 1 component.
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Musculoskeletal

Courage Therapeutics and University of Michigan divulge new MC4R agonists and MC3R antagonists/partial agonists

March 23, 2023
Courage Therapeutics Inc. and University of Michigan have synthesized peptides acting as melanocortin MC4 receptor (MC4R) agonists and/or MC3 receptor (MC3R) antagonist/partial agonists reported to be useful for the treatment of arthritis, depression, diabetes, hypertension, renal disorders and sleep apnea.
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Giant plasma membrane vesicles
Musculoskeletal

Deficiencies in membrane lipid biosynthesis cause muscle degeneration

March 23, 2023
By Subhasree Nag
A research team led by Domagoj Cikes at the Institute of Molecular Biotechnology of the Austrian Academy of Sciences (IMBA) and Josef Penninger at the University of British Columbia has discovered the critical role of the enzyme phosphate cytidylyltransferase 2 (PCYT2) in muscle health. Their findings appeared in the March 20, 2023, online edition of Nature Metabolism.
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Musculoskeletal

Shanghai Visonpharma discovers new TLR7 and TLR8 antagonists

March 22, 2023
Shanghai Visonpharma Co. Ltd. has described Toll-like receptor 7 (TLR7) and/or TLR8 antagonists reported to be useful for the treatment of multiple sclerosis, lupus nephritis, psoriasis, rheumatoid arthritis, systemic scleroderma (systemic sclerosis), Sjogren syndrome and systemic lupus erythematosus.
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Newborn baby feet and DNA base pair letters A, T, C and G.
Biomarkers

New pathogenic variant in NEB confirms diagnosis of nemaline myopathy 2

March 21, 2023
Researchers from the University of Utah applied RNASeq analysis for an undiagnosed case of a critically ill newborn with a complex phenotype, with the aim of providing better diagnosis and improving treatment outcomes.
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