The largest analysis to date of patients taking GLP-1 receptor agonists (GLP-1RAs) has investigated their effects on nearly 175 diseases, and found that compared to three other classes of diabetes medications, individuals with a prescription for GLP-1RAs had a reduced risk of 42 diseases, and an increased risk of 19. The findings, which were published Jan. 20, 2025, in Nature Medicine, provide a comprehensive overview of GLP-1RAs’ effects.

Having 35 copies of the CAG triplet in the gene that causes Huntington’s disease is not a problem. Inheriting 40 could be a sign that goes unnoticed for decades, until reaching 80. From there, the process accelerates and neural death occurs when reaching 150 repeats. Huntington’s disease neurodegeneration is not determined by what, but by how much, according to a study conducted at the Broad Institute.

T-cell leukemia homeobox protein 3 (TLX3) is a key regulator of fate specification of excitatory neurons and found expressed in proliferating granule neuron progenitors (GNPs) of the cerebellum, but its main role is not well known.

Kyorin Pharmaceutical Co. Ltd. has entered into an option agreement with Biodol Therapeutics SAS.

Pheno Therapeutics Ltd. has received clinical trial authorization (CTA) from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for its lead candidate, PTD-802. The program will progress to a first-in-human phase I trial.

Ips Heart Inc. has been awarded orphan drug designation by the FDA for its GIVI-MPC stem cell therapy for Becker muscular dystrophy. GIVI-MPC has the unique ability to create new muscle with full length dystrophin in Becker muscular dystrophy.

Recent work proposes a promising therapeutic approach for dysferlinopathies.