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BioWorld - Tuesday, April 7, 2026
Home » Topics » Disease categories and therapies » Musculoskeletal

Musculoskeletal
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Chromosome with shortened telomeres
Neurology/Psychiatric

TRF2 prevents telomere shortening and prolongs cardiomyocyte survival in DMD

Feb. 3, 2023
The lack of dystrophin causes Duchenne muscular dystrophy (DMD), a muscle-wasting disease that is often accompanied by heart failure due to cardiomyocyte death and fibrosis that can lead to death of the patient. It has been proven that telomere shortening is a hallmark of DMD cardiomyocytes. Researchers from the Stanford School of Medicine have recently investigated whether preventing telomere shortening and attrition may be a therapeutic approach in DMD.
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Joint pain illustration
Biomarkers

A 13-protein marker panel may predict osteoarthritis progression

Feb. 3, 2023
Osteoarthritis (OA) is the most common joint disease and a leading cause of disability worldwide. Its treatment is still elusive due to difficulties with early diagnoses and patient risk identification. This leads to a need for reliable biomarkers for OA prognosis or to identify patients at risk of progression.
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Musculoskeletal

Dice Alpha patents new IL-17A production inhibitors

Feb. 2, 2023
Dice Alpha Inc. has synthesized phenyl acetamide compounds acting as interleukin-17A (IL-17A) production inhibitors reported to be useful for the treatment of psoriasis, radiographic axial spondyloarthritis (ankylosing spondylitis), hidradenitis suppurativa, spondyloarthritis, psoriatic and rheumatoid arthritis.
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Stem cells
Newco news

Juvena looking to hESC-secreted proteins for treating age-related disease

Jan. 11, 2023
By Jennifer Boggs
Juvena Therapeutics Inc. co-founder and CEO Hanadie Yousef had the company’s name picked out several years before it was officially incorporated in 2017 to combine Yousef’s work in the mechanics of aging with an underutilized class of biologics and an advanced proteomics platform to tackle chronic and age-related diseases.
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Arthritis pain illustration
Inflammatory

New approach to osteoarthritis identified through GWAS

Jan. 11, 2023
By Nuala Moran
An Icelandic genome-wide association study that linked variants in a gene which regulates retinoic acid synthesis to severe osteoarthritis (OA) of the hands, has led on to the discovery of an anti-inflammatory role for the vitamin A metabolite and pointed to a new class of potentially disease-modifying drugs. A proof-of-concept clinical trial of talarozole, a retinoic acid metabolism blocking agent, is now taking place to assess if increasing retinoic acid production suppresses inflammation in the joint tissues of patients with OA.
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Comparison of senescent cells in regenerating muscle.
Musculoskeletal

Senescent cells are toxic to their neighbors, prevent muscle regeneration

Dec. 22, 2022
By Mar de Miguel
The first in vivo cell atlas of senescent tissue in skeletal muscle has identified the damaging properties of these cells and explained why they block muscle regeneration. According to a study at Pompeu Fabra University led by scientists from Altos Labs Inc., cell damage caused the senescence of the cells, which secreted toxic substances into the surrounding microenvironment, causing fibrosis and preventing tissue regeneration.
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3D rendering showing osteoporosis in the femur
Musculoskeletal

SIK2/3 inhibition promotes bone formation in mice

Dec. 20, 2022
To date, there are no current orally available compounds that promote bone formation for treating osteoporosis; most treatments act by inhibiting osteoclastic bone resorption, leading to increased bone mineral density and reduced hip fracture rate in a modest way. Analogues of parathyroid hormone (PTH) are the most frequently used bone anabolic agents, which even though effective, they require daily injections.
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Genetic/Congenital

Role of SOX9 and SOX10 in mouse model of campomelic dysplasia

Dec. 19, 2022
The sex-determining region Y (SRY)-related HMG-box, group E (SOXE) transcription factors SOX9 and SOX10 are essential for the specification and differentiation of many progenitor cell types and for the development of several organs and tissues.
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Avidity muscles into positive phase I/II data with lead candidate AOC-1001

Dec. 14, 2022
By Lee Landenberger
A preliminary assessment of Avidity Biosciences Inc.’s phase I/II study of AOC-1001 in myotonic dystrophy type 1 (DM1) showed the first-ever targeted delivery of RNA into muscle, an area previously untreatable with existing RNA therapeutics. Sarah Boyce, Avidity’s CEO, said in a Dec. 14 call that the antibody oligonucleotide conjugate’s data were unprecedented in the RNA space and in myotonic dystrophy type 1 (DM1), labeling it a “revolutionary advancement.”
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Musculoskeletal

Janssen Pharmaceutica presents new NLRP3 inflammasome inhibitors

Nov. 24, 2022
Janssen Pharmaceutica NV has divulged NLRP3 inflammasome inhibitors reported to be useful for the treatment of osteoarthritis, cancer, nonalcoholic steatohepatitis (NASH), skin lesion, sarcoidosis, as well as cardiovascular, metabolic and Alzheimer's diseases, among others.
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