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BioWorld - Wednesday, February 11, 2026
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Neurology/psychiatric

New canine model of Duchenne muscular dystrophy reported

March 24, 2025
Researchers from Suzhou Genassist Therapeutics Co. Ltd. have developed a novel canine model of Duchenne muscular dystrophy (DMD), expected to be more predictive of disease pathogenesis and treatment efficacy.
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Close up of hand scratching arm with psoriasis patches
Dermatologic

Discovery of potent and orally bioavailable IL-17A inhibitors for the treatment of psoriasis

March 21, 2025
Researchers from Anew Therapeutics Pte Ltd. recently detailed the discovery of novel oral IL-17A small-molecule inhibitors as candidates for the treatment of psoriasis.
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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.
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Biomarkers

New findings relate MAD1L1 gene with mosaic variegated aneuploidy syndrome

March 21, 2025
Mosaic variegated aneuploidy syndrome (MVAS) is an autosomal recessive disorder characterized by mosaic aneuploidy. Its clinical manifestations include growth and developmental delay, congenital malformations and increased cancer risk. Genetic variants involved in MVAS affect the chromosomal segregation during mitosis, where individuals often show mosaicism and chromosomal instability.
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Toilets could be the next big health device: Medical Korea 2025

March 20, 2025
By Marian (YoonJee) Chu
Seung-min Park, professor at Nanyang Technological University and cofounder of Kanaria Health, is working to manufacture smart bidets that can capture biomarker data from urine and stool automatically and enable continuous monitoring.
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Dermatologic

NOA-104 ameliorates atopic dermatitis in the preclinical setting

March 20, 2025
Atopic dermatitis (AD) is mainly triggered by immune dysregulation, barrier dysfunction and inflammation propagation, and chronic itching increases the susceptibility to infections.
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Muscular dystrophy
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).
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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).
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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.
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Ocular

Nonclinical analysis of the hyperthermic effect of SJP-0132

March 19, 2025
Researchers from Senju Pharmaceutical Co. Ltd. presented results from preclinical studies that assessed the hyperthermic effects of SJP-0132, a selective antagonist of transient receptor potential vanilloid 1 (TRPV1), which is currently awaiting approval in Japan as an ophthalmic treatment to alleviate the signs and symptoms of dry eye disease.
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