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Home » Topics » Disease categories and therapies » Musculoskeletal

Musculoskeletal
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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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Celltrion wins FDA approval of denosumab, omalizumab biosimilars

March 18, 2025
By Marian (YoonJee) Chu
Celltrion Inc. is on a biosimilar roll with the U.S. FDA this month, having gained clearance of Stoboclo and Osenvelt as products referencing Amgen Inc.’s biologic, denosumab (Prolia, Xgeva), along with Omlyclo becoming the first and only interchangeable biosimilar to omalizumab (Xolair, Genentech Inc. and Novartis AG).
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Stock table, gold arrow pointing down

CMV history cited in DMD liver death on Sarepta’s Elevidys

March 18, 2025
By Randy Osborne
Wall Street was weighing the gravity of the death from acute liver failure of a patient who was treated for Duchenne muscular dystrophy (DMD) with Sarepta Therapeutics Inc.’s gene therapy, Elevidys (delandistrogene moxeparvovec). Liver injury is a known possible side effect of the product, first approved by the U.S. FDA in June 2023 for DMD, as well as other AAV-mediated gene therapies, and the potential problem is highlighted in Elevidys’ prescribing information.
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Closeup of wheelchair

Avidity eyes 2025 BLA on back of positive DMD data

March 17, 2025
By Jennifer Boggs
While data on functional endpoints are still to come, Avidity Biosciences Inc. executives said the firm is moving ahead with plans for a BLA filing by the end of 2025 for del-zota, an antibody-oligonucleotide conjugate, in Duchenne muscular dystrophy with mutations amenable to exon 44 skipping (DMD44), based on positive top-line data that analysts say bode well for Avidity’s other late-stage programs targeting rare neuromuscular diseases.
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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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Stock merger illustration

Endo the line: Mallinckrodt merger worth $6.7B

March 13, 2025
By Randy Osborne
The pairing of Mallinckrodt plc and Endo Inc. is expected to create a pharma heavyweight that will generate $3.6 billion in 2025 revenue after the duo combines their generic pharmaceuticals businesses and Endo’s sterile injectables setup. Terms call for Dublin-based Endo shareholders to collect $80 million in cash and own 49.9% of the combined firm. Owners of stock in Mallinckrodt will hold the rest of the new entity in the arrangement, which bears an enterprise value of $6.7 billion and is expected to close in the second half of this year.
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