Musculoskeletal - Articles - Page 6

Genetic/congenital

CMT2E murine model reveals early axonal damage

March 21, 2025

Charcot-Marie-Tooth disease type 2E (CMT2E) is a slow and progressive neuropathy characterized by axonal dysfunction. Its clinical phenotype includes muscle weakness and atrophy, sensory loss and reduced nerve conduction velocity, among others.

Neurology/psychiatric

Insmed reports preclinical data on INS-1201 for DMD

March 20, 2025

Investigators from Insmed Inc. have presented new preclinical data on the efficacy of their adenoviral vector (AAV9)-based gene therapy INS-1201 for the treatment of Duchenne muscular dystrophy (DMD).

Neurology/psychiatric

DMD base editing candidate shows safety and efficacy in preclinical models

March 20, 2025

At this week’s Muscular Dystrophy Association Clinical and Scientific Conference in Dallas, researchers from Suzhou Genassist Therapeutics Co. Ltd. presented preclinical data for GEN-6050X (ss.AAV9.oTAM and ss.AAV9.hE50-sgRNA).

3D cross-section illustration of muscle anatomy

Musculoskeletal

Epirium Bio’s first-in-class 15-PGDH inhibitor restores muscle strength in aged mice

March 19, 2025

Sarcopenia is an age-related condition in which muscle mass and strength decrease, leading to reduced overall physical performance. As fast-twitch muscle fibers are more impacted by age-related decline, strategies aimed at enhancing the regeneration and functionality of these fibers are essential to prevent the progression of sarcopenia.

Illustration of man holding magnifying glass to human body model showing muscle anatomy

Neurology/psychiatric

New in vitro model of inclusion body myositis based on human iPSC-myotubes

March 17, 2025

Inclusion body myositis (IBM) is an inflammatory myopathy causing proximal and distal muscle weakness. IBM’s cause remains unknown, lacking validated models, biomarkers and effective treatment strategies. Histopathological studies identified inflammatory infiltrates, rimmed vacuoles, and mitochondrial changes in the muscles of IBM patients.

Inflammatory

Inflammation appears to cause mutations linked to MS progression

March 10, 2025

By Tamra Sami

For the first time, researchers have identified that inflammation – long associated with multiple sclerosis (MS) – appears to cause increased mutations that damage neurons linked to MS progression. Researchers at the Florey Institute and the University of Melbourne studied MS brain lesions, which are areas of past or ongoing brain inflammation that are visible as spots on MRI scans.

Musculoskeletal

Roche patents new SIK inhibitors

Feb. 14, 2025

F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have identified new benzimidazoles acting as serine/threonine-protein kinase SIK inhibitors reported to be useful for the treatment of rheumatoid arthritis, metabolic dysfunction-associated steatohepatitis (MASH), giant cell arteritis, primary sclerosing cholangitis, inflammatory bowel disease, atherosclerosis, type 2 diabetes and glomerulonephritis, among others.

Musculoskeletal

Bristol Myers Squibb discloses new PAD4 inhibitors for rheumatoid arthritis

Feb. 12, 2025

Bristol Myers Squibb Co. has patented new protein-arginine deiminase type-4 (PADI4; PAD4) inhibitors reported to be useful for the treatment of rheumatoid arthritis, among others.

3D rendering showing osteoporosis in the femur

Endocrine/metabolic

Pyrimidine derivative could help fight secondary osteoporosis

Jan. 31, 2025

Glucocorticoid-induced osteoporosis, the leading cause of secondary osteoporosis, is characterized by diminished bone density and compromised osteoblast function. As current treatment options often have significant side effects, researchers are actively seeking new drug candidates to improve patient outcomes.

Endocrine/metabolic