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Home » Topics » Disease categories and therapies » Musculoskeletal

Musculoskeletal
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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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Alteogen headquarters in Daejeon, South Korea

Alteogen merges two subsidiaries, forming Alteogen Biologics

May 27, 2025
By Marian (YoonJee) Chu
No Comments
Alteogen Inc. completed a merger between two subsidiaries – Altos Biologics Inc. and Alteogen Healthcare Inc. – branding the new entity as Alteogen Biologics Inc.
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Alteogen headquarters in Daejeon, South Korea

Alteogen merges two subsidiaries, forming Alteogen Biologics

May 23, 2025
By Marian (YoonJee) Chu
No Comments
Alteogen Inc. completed a merger between two subsidiaries – Altos Biologics Inc. and Alteogen Healthcare Inc. – branding the new entity as Alteogen Biologics Inc.
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Photo of older woman holding hand that's colored red
Dermatologic

Strong JAK1-selective inhibitor IN-115314: pharmacokinetics and -dynamics in dogs

May 23, 2025
No Comments
Rheumatoid arthritis, atopic dermatitis, ulcerative colitis and several other inflammatory diseases involve hyperactivation of JAK-STAT signaling, in which the kinase JAK binds and phosphorylates the transcription factor STAT, which then turns on gene expression.
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Annji's phase I/II positive in spinal, bulbar muscular atrophy

May 22, 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.
Read More

Apimeds Pharmaceuticals raises $13.5M from NYSE American debut

May 20, 2025
By Marian (YoonJee) Chu
No Comments
Apimeds Pharmaceuticals US Inc. (APUS), a subsidiary of Kospi-listed Inscobee Inc., said May 12 that it raised $13.5 million from its stock sale on the NYSE American exchange May 9 through an offering of 3.375 million shares at $4 per share. Hopewell, N.J.-based APUS is the second pharmaceutical company with Asian ties to float shares on the U.S. market this year.
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