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Home » Topics » Disease categories and therapies » Musculoskeletal

Musculoskeletal
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Boy in wheelchair, scenic overlook
Musculoskeletal

SAT-3247 adds rare pediatric disease designation

Aug. 8, 2024
After receiving Orphan Drug Designation earlier this year, Satellos Bioscience Inc. announced that the FDA has granted Rare Pediatric Disease Designation to SAT-3247 for the potential treatment of Duchenne muscular dystrophy (DMD).
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Musculoskeletal

Cogent Biosciences discovers new FGFR inhibitors

Aug. 7, 2024
Cogent Biosciences Inc. has disclosed compounds acting as fibroblast growth factor receptor (FGFR) inhibitors reported to be useful for the treatment of achondroplasia, cancer, craniosynostosis, Alzheimer's disease, fibrosis, pulmonary fibrosis, systemic scleroderma (systemic sclerosis) and thanatophoric dysplasia, among others.
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Musculoskeletal

Caregen describes new peptide for cartilage regeneration

Aug. 6, 2024
Caregen Co. Ltd. has identified peptides reported to be useful for the treatment of cartilage injury, fracture, intervertebral disc herniation, degenerative intervertebral disc disorder, osteoarthritis, osteomalacia and muscular injury.
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Musculoskeletal

Z-526 alleviates chemotherapy-induced cachexia muscle atrophy in vivo

Aug. 2, 2024
Researchers from East China Normal University and Fudan University presented the discovery of a new oral compound, Z-526, being developed as a treatment to alleviate chemotherapy-induced cachectic muscle loss.
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Stock chart, red down arrow

Mayday, skipper: Pepgen shares run aground on DMD phase II data

July 31, 2024
By Randy Osborne
Pepgen Inc. fell slightly short of its phase II dystrophin goal with PGN-EDO51 for patients with Duchenne muscular dystrophy (DMD) whose mutations are amenable to an exon 51-skipping approach, but Wall Street reacted in a big way, sending the Boston-based firm’s stock (NASDAQ:PEPG) down 33%, or $5.55, to close July 31 at $11.43.
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Joint pain illustration
Musculoskeletal

STX-0119 shows therapeutic potential in models of osteoarthritis

July 31, 2024
Researchers from the Second Affiliated Hospital of Fujian Medical University and Shaoxing People's Hospital presented preclinical data for the signal transducer and activator of transcription 3 (STAT3) inhibitor STX-0119, which has been previously validated in models of cancer.
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Leqembi

EMA’s CHMP issues thumbs-down on Alzheimer’s drug Leqembi

July 30, 2024
By Nuala Moran
The EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended approval of 14 drugs and the extension of the label of 11 others at its July meeting, but, inevitably, it was the decision to turn down the Alzheimer’s disease therapy Leqembi (lecanemab) that stirred the greatest reaction.
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Businessman and IPO virtual screen

Jiuyuan, Biokin, Transthera file for Hong Kong IPOs in July

July 30, 2024
By Marian (YoonJee) Chu
Three Chinese biopharmaceuticals have filed for IPOs on the Hong Kong exchange in July alone, highlighting a potential “newfound positivity” to strengthen the HKEX IPO market in the second half of 2024.
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Musculoskeletal

Tibet Haisco Pharmaceutical identifies MYH2 inhibitors for muscular dystrophy

July 30, 2024
Tibet Haisco Pharmaceutical Co. Ltd. has divulged myosin-2 (MYH2) inhibitors reported to be useful for the treatment of muscular dystrophy.
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Bone marrow illustration with doctor background
Endocrine/metabolic

ENTR1 inhibition alleviates lipid-related bone marrow problems

July 24, 2024
Researchers from Shandong University and affiliated organizations have presented data from a study that aimed to assess the role of endosome-associated trafficking regulator 1 (ENTR1) in adipogenesis.
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