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Home » Topics » Disease categories and therapies » Musculoskeletal

Musculoskeletal
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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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IPO money

With warming market, Hinge swings back to IPO

March 13, 2025
By Annette Boyle
After scrapping plans for an IPO in 2022, Hinge Health Inc. is taking a more favorable view of going public given the increased support seen for health care and med-tech stocks in recent months. The San Francisco-based company, a digital provider of physical therapy services, filed a form S-1 with the U.S. SEC stating it plans to offer an unspecified number of shares of class A common stock on the New York Stock Exchange this year.
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Celltrion wins FDA approval of denosumab, omalizumab biosimilars

March 12, 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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Justin Rubio working in the lab
Inflammatory

Inflammation appears to cause mutations linked to MS progression

March 11, 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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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.
Read More
3D illustration of transparent human torso with close up of spinal cord

Qyuns’ QX-002N hits endpoints in ankylosing spondylitis trial

Feb. 25, 2025
By Tamra Sami
Qyuns Therapeutics Co. Ltd.’s monoclonal antibody targeting IL-17A, QX-002N, met both primary and secondary endpoints in a phase III trial in ankylosing spondylitis.
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Delivery of therapeutic oligonucleotide cargoes into tissues and cells

Pepgen rallies as early data suggest sporting chance in DM1

Feb. 24, 2025
By Randy Osborne
Pepgen Inc. seems to have gained a leg up on competitors in early data with PGN-EDODM1 in myotonic dystrophy type 1 (DM1), and shares of the Boston-based firm (NASDAQ:PEPG) closed Feb. 24 at $2.29, up 92 cents, or about 67%. The company unveiled initial positive data from the 5- and 10-mg/kg dose cohorts in the ongoing Freedom-DM1 phase I study with PGN-EDODM1, which deploys Boston-based Pepgen’s Enhanced Delivery Oligonucleotide technology to deliver a therapeutic oligonucleotide that is designed to restore the normal function of MBNL1, a key RNA splicing protein.
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Solid rocks DMD space with phase I/II gene therapy data

Feb. 18, 2025
By Randy Osborne
Solid Biosciences Inc. is preparing for a sit-down with the U.S. FDA this year to discuss the firm’s results with the next-generation gene therapy SGT-003 for Duchenne muscular dystrophy (DMD).
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