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BioWorld - Thursday, April 16, 2026
Home » Topics » Disease categories and therapies » Musculoskeletal

Musculoskeletal
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3d illustration of human body muscle tissue anatomy
Musculoskeletal

New mouse model of congenital fiber type disproportion

March 7, 2024
Congenital fiber type disproportion (CFTD) is a disorder characterized by the atrophy of slow-twitch type 1 muscle fibers.
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Drug vials and syringe

Sandoz’s denosumab biosimilars challenge Amgen with US FDA nod

March 6, 2024
By Lee Landenberger
The biosimilars revolution continues with the U.S. FDA’s approval of the first denosumab biosimilars: Wyost (denosumab-bbdz) and Jubbonti (denosumab-bbdz) from Sandoz Inc. for treating osteoporosis and to prevent bone problems in cancer. The approval puts up a strong challenge to Amgen Inc.’s Prolia, the first biologic for osteoporosis, and Xgeva, for bone cancer.
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Light micrograph of skeletal muscle.
Neurology/Psychiatric

MP-1032 shows beneficial effects at treating murine DMD

March 6, 2024
The long-term use of corticosteroids for treating Duchenne muscular dystrophy (DMD) is tied to several undesired effects, compromising the patient’s quality of life; hence, the use of nonsteroidal drugs is highly desirable in the treatment of DMD. Metriopharm AG is investigating the nonsteroidal drug MP-1032 for DMD and recently presented data from studies in a model of DMD.
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Illustration of motor neuron connecting to muscle fiber

Pivotal trial next for Pharmaust after MND/ALS phase I success

March 5, 2024
By Tamra Sami
Pharmaust Ltd.’s monepantel met its primary safety endpoints and showed positive signals of potential efficacy in a phase I trial in patients with motor neuron disease (MND)/amyotrophic lateral sclerosis (ALS). With these results, the company will now progress to a pivotal phase II/III trial by midyear, Pharmaust CEO Michael Thurn told BioWorld.
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DNA illustration
Biomarkers

First susceptibility gene for rare form of psoriatic arthritis identified

March 1, 2024
Psoriatic arthritis mutilans (PAM) is a rare variant and the most severe form of psoriatic arthritis where patients present erosions of the small joints and osteolysis leading to joint disruption and shortening of one or more digits. To date, no susceptibility genes have been determined.
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Illustration of motor neuron connecting to muscle fiber

Pivotal trial next for Pharmaust after MND/ALS phase I success

Feb. 29, 2024
By Tamra Sami
Pharmaust Ltd.’s monepantel met its primary safety endpoints and showed positive signals of potential efficacy in a phase I trial in patients with motor neuron disease (MND)/amyotrophic lateral sclerosis (ALS). With these results, the company will now progress to a pivotal phase II/III trial by midyear, Pharmaust CEO Michael Thurn told BioWorld.
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3D dollar sign

Kyowa pays $100M up front for Bridgebio’s infigratinib in Japan

Feb. 20, 2024
By Marian (YoonJee) Chu
Palo Alto, Calif.-based Bridgebio Pharma Inc. will hand over development and sales of its rare bone growth disorder therapy, infigratinib, in Japan to Kyowa Kirin Co. Ltd. under its latest exclusive licensing deal.
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Musculoskeletal

Genescience Pharmaceuticals discovers new cannabinoid CB2 receptor agonists

Feb. 16, 2024
Genescience Pharmaceuticals Co. Ltd. has described cannabinoid CB2 receptor agonists reported to be useful for the treatment of arthritis, systemic lupus erythematosus, inflammatory bowel disease, allergic contact dermatitis, colitis, autoimmune diseases and pain.
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3D rendering showing osteoporosis in the femur
Biomarkers

New SNP in PAPSS2 gene tied to higher osteoporosis risk

Feb. 15, 2024
Genome sequencing has identified several single-nucleotide polymorphisms (SNPs) tied to osteoporosis, but most of them are located in noncoding regions. Investigators identified a SNP which impacted the YY2-PAPSS2 axis and risk of osteoporosis; the PAPSS2 gene encodes bifunctional 3’-phosphoadenosine 5’-phosphosulfate synthase 2.
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Biomarkers

New COL3A1 mutation found in case of bleeding of unknown cause

Feb. 12, 2024
Bleeding of unknown cause is a group of rare disorders that are still difficult to accurately diagnose. A case report on a patient with hematoma in the perineal region after her first delivery was presented.
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