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BioWorld - Sunday, February 15, 2026
Home » Topics » Disease categories and therapies » Musculoskeletal

Musculoskeletal
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Bristol Myers Squibb sign
ACS Fall 2022

Evolution of carbazole core as TLR7/8 antagonists

Sep. 2, 2022
Structure-guided design for carbazole antagonists of Toll-like receptors 7 and 8 (TLR7/8) for the potential treatment of autoimmune disorders was reported by Bristol Myers Squibb Co. TLR activation can induce proinflammatory pathway activation, which has been identified in patients with lupus.
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BeiGene presents data on selective BTK inhibitor BGB-8035

Aug. 26, 2022
Researchers from BeiGene presented data on the discovery of highly selective covalent tyrosine-protein kinase BTK inhibitors for the potential treatment of B-cell malignancies and autoimmune disorders.
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Pain illustration

Not good enough? Blueprint slides on positive top-line data in non-advanced SM

Aug. 17, 2022
By Jennifer Boggs
“No good data goes unpunished in this market,” H.C. Wainwright analyst Andrew Fein wryly noted in an Aug. 17 research report highlighting Wall Street’s dismal response to Blueprint Medicines Corp.’s positive top-line readout of the registrational Pioneer study, in which KIT inhibitor Ayvakit (avapritinib) met the primary and all key secondary endpoints in patients with non-advanced systemic mastocytosis.
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Silhouette of person walking with a cane

Kringle Pharma’s phase II ALS study falls short

Aug. 16, 2022
By Tamra Sami
Kringle Pharma Inc.’s phase II trial evaluating its recombinant human hepatocyte growth factor ligand, oremepermin alfa, failed to meet both primary and secondary endpoints in a study of its potential to help people with amyotrophic lateral sclerosis (ALS).
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Kringle Pharma’s phase II ALS study falls short

Aug. 15, 2022
By Tamra Sami
Kringle Pharma Inc.’s phase II trial evaluating its recombinant human hepatocyte growth factor ligand, oremepermin alfa, failed to meet both primary and secondary endpoints in a study of its potential to help people with amyotrophic lateral sclerosis (ALS).
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New MRGPRX2 antagonists identified at GlaxoSmithKline

Aug. 10, 2022
GlaxoSmithKline has divulged mas-related G protein-coupled receptor member X2 (MRGPRX2) antagonists reported to be useful for the treatment of osteoarthritis, migraine, asthma, irritable bowel syndrome, atopic dermatitis, psoriasis, urticaria and rheumatoid arthritis, among other disorders.
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JMJD3 epigenetically regulates osteoarthritis progression: a potential therapeutic target

Aug. 9, 2022
In a recent study, researchers from Shanghai Jiao Tong University in China investigated the epigenetic program responsible for cartilage injury induced by aberrant mechanical force, with the final aim of exploring a potential epigenetic-based therapy approach for osteoarthritis.
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Targeting IL-6 signaling alleviates PTOA-associated cartilage degeneration and pain

Aug. 5, 2022
Post-traumatic OA (PTOA) is a specific type of osteoarthritis (OA) caused by injury. Both OA and PTOA are caused by an imbalance in catabolic and anabolic processes in articular cartilage as well as proinflammatory changes throughout the joint, which lead to joint degeneration and pain.
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Good news for Amylyx as it prepares for adcom redux

Aug. 4, 2022
By Mari Serebrov
Amylyx Pharmaceuticals Inc. got good news when the Institute for Clinical and Economic Review posted a revised evidence report Aug. 4 that assessed the comparative clinical effectiveness and value of the company’s AMX-0035 and Mitsubishi Tanabe Pharma America Inc.’s Radicava (edaravone) in treating amyotrophic lateral sclerosis.
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Sarepta shares jump as it plots fast FDA review for DMD gene therapy

July 29, 2022
By Richard Staines
Sarepta Therapeutics Inc. said it plans to file a BLA for its gene therapy for Duchenne muscular dystrophy (DMD), SRP-9001, with the U.S. FDA, potentially setting up a decision in the first half of 2023 for the therapy developed in partnership with Switzerland’s Roche Holding AG. The Cambridge, Mass.-based biotech said the BLA will seek accelerated approval for the therapy, also known as delandistrogene moxeparvovec, for ambulant individuals with DMD.
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