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BioWorld - Thursday, December 18, 2025
Home » Topics » Disease categories and therapies » Musculoskeletal

Musculoskeletal
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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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Musculoskeletal

Roche patents new SIK inhibitors

Feb. 14, 2025
F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have identified new benzimidazoles acting as serine/threonine-protein kinase SIK inhibitors reported to be useful for the treatment of rheumatoid arthritis, metabolic dysfunction-associated steatohepatitis (MASH), giant cell arteritis, primary sclerosing cholangitis, inflammatory bowel disease, atherosclerosis, type 2 diabetes and glomerulonephritis, among others.
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Musculoskeletal

Bristol Myers Squibb discloses new PAD4 inhibitors for rheumatoid arthritis

Feb. 12, 2025
Bristol Myers Squibb Co. has patented new protein-arginine deiminase type-4 (PADI4; PAD4) inhibitors reported to be useful for the treatment of rheumatoid arthritis, among others.
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Central nervous system

Alterity’s ATH-434 looks to be disease modifying in multiple system atrophy

Feb. 4, 2025
By Tamra Sami
Alterity Therapeutics Ltd. reported positive top-line phase II results for lead candidate ATH-434 for treating multiple system atrophy, a rare neurological disorder similar to Parkinson's disease.
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DMD space takes heart from Cumberland’s phase II ifetroban data

Feb. 4, 2025
By Randy Osborne
The first successful phase II trial in Duchenne muscular dystrophy (DMD) heart disease rolled out from Cumberland Pharmaceuticals Inc., which said top-line findings from the experiment called Fight DMD showed promising results in the indication that represents the main cause of death for such patients.
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3D rendering showing osteoporosis in the femur
Endocrine/metabolic

Pyrimidine derivative could help fight secondary osteoporosis

Jan. 31, 2025
Glucocorticoid-induced osteoporosis, the leading cause of secondary osteoporosis, is characterized by diminished bone density and compromised osteoblast function. As current treatment options often have significant side effects, researchers are actively seeking new drug candidates to improve patient outcomes.
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Central nervous system

Alterity’s ATH-434 looks to be disease modifying in multiple system atrophy

Jan. 30, 2025
By Tamra Sami
Alterity Therapeutics Ltd. reported positive top-line phase II results for lead candidate ATH-434 for treating multiple system atrophy, a rare neurological disorder similar to Parkinson's disease.
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Endocrine/metabolic

Japan Tobacco divulges PDE7A inhibitor for osteoporosis prevention

Jan. 28, 2025
Due to the widespread presence of PDE7 in various tissues, the development of PDE7 inhibitors has been limited by potential adverse side effects.
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Veru weight loss study hits primary endpoint, stock drops

Jan. 27, 2025
By Lee Landenberger
Positive data for Veru Inc.’s enobosarm lend more weight to the potential progress of the company’s body-mass preservation program in patients taking Wegovy (semaglutide). The side effect of lean mass loss has dogged those taking GLP-1s. The study results didn’t support the company’s stock on the day of the data release as it had the previous four weeks.
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Musculoskeletal

Xi’an Xintong Medicine Research divulges new JAK3 inhibitors

Jan. 27, 2025
Xi’an Xintong Medicine Research Co. Ltd. has synthesized tyrosine-protein kinase JAK3 inhibitors reported to be useful for the treatment of rheumatoid arthritis, alopecia areata, vitiligo, asthma, chronic obstructive pulmonary disease, cancer and inflammatory bowel disease.
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