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Home » Topics » Disease categories and therapies » Musculoskeletal

Musculoskeletal
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Test tubes, dropper and capsules
Musculoskeletal

SAN-523 demonstrates anti-inflammatory efficacy in preclinical models of microcrystalline arthropathies

June 18, 2025
No Comments
University of Lausanne has reported promising preclinical data on SAN-523, a first-in-class positive allosteric modulator of cystathionine gamma-lyase (CSE), developed as a potential therapeutic for microcrystalline arthropathies, including gout and calcium pyrophosphate deposition (CPPD) disease.
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Dyne vHOT in DM1 but shares cool on endpoint switch

June 17, 2025
By Randy Osborne
No Comments
The revised trial protocol that means a delay in filing for U.S. approval of DYNE-101 to treat myotonic dystrophy type 1 (DM1) dented shares of Dyne Therapeutics Inc. (NASDAQ:DYN), which closed June 17 at $10.86, down $2.96, or 21%.
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Musculoskeletal

New tracer has diagnostic utility for rheumatoid arthritis

June 17, 2025
No Comments
Rheumatoid arthritis (RA) is a chronic autoimmune disease characterized by inflammation and damage of the joints. PET/CT imaging is a useful tool for early disease assessment and monitoring treatment efficacy.
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Sarepta signage

Elevidys’ second liver death hits Sarepta, sends DMD ripples

June 16, 2025
By Randy Osborne
No Comments
How the U.S. FDA might respond became a serious question for Wall Street as Sarepta Therapeutics Inc. made known a second death due to acute liver failure with gene therapy Elevidys (delandistrogene moxeparvovec), cleared for Duchenne muscular dystrophy (DMD). Shares of Cambridge, Mass.-based Sarepta (NASDAQ:SRPT) closed June 16 at $20.94, down $15.24, or 42%, as Wall Street digested the news.
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Close up of senior man holding wrist of arthritic hand
Musculoskeletal

CXCR7 agonist to mitigate rheumatoid arthritis

June 16, 2025
No Comments
Researchers at Ileadbms Co. Ltd. have announced promising results of their novel drug IL-21120033 in a preclinical model of rheumatoid arthritis. The drug acts as a selective agonist of the chemokine receptor CXCR7.
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DNA mutations or genetic disorder concept art

Regenxbio’s Duchenne gene therapy data positive as shares falter

June 5, 2025
By Lee Landenberger
No Comments
Regenxbio Inc.’s gene therapy in treating Duchenne muscular dystrophy (DMD) produced positive initial phase I/II results from its first five patients. However, the company’s stock (NASDAQ:RGNX) shuddered on June 5 as shares closed at $8.36 each, a drop of 17% on the day.
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Musculoskeletal

Inflamax Pharmaceuticals patents new PACE4 inhibitors for osteoarthritis

June 2, 2025
Inflamax Pharmaceuticals (Guangzhou) Co. Ltd. has disclosed proprotein convertase subtilisin/kexin-type 6 (PCSK6; PACE4; PC7A) inhibitors reported to be useful for the treatment of osteoarthritis.
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Mitochondria

CRL blocks Barth syndrome drug; Stealth aims to try again

May 29, 2025
By Karen Carey
No Comments
Developing a therapy for an ultra-rare condition has its challenges, including finding enough patients for clinical enrollment and convincing regulatory authorities that limited data prove the candidate is safe and effective. For that reason, Stealth Biotherapeutics Inc. has faced numerous roadblocks getting its mitochondria-targeting elamipretide across the finish line for Barth syndrome, a condition that affects about 230 to 250 males worldwide, including fewer than 150 in the U.S.
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Musculoskeletal

Novel BAG3-expressing gene therapy shows promise for IBM

May 29, 2025
No Comments
Inclusion body myositis (IBM) is the most common acquired myopathy in people over 50 years of age, characterized by chronic and progressive muscle weakness, where its pathogenesis involves inflammatory and degenerative pathways that are not well understood to date.
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Annji's phase I/II positive in spinal, bulbar muscular atrophy

May 27, 2025
By Tamra Sami
No Comments
Annji Pharmaceutical Co. Ltd.’s rosolutamide (AJ-201, JM-17) achieved positive results in a phase I/II trial in adults with spinal and bulbar muscular atrophy, also known as Kennedy’s disease.
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