About 10% of amyotrophic lateral sclerosis (ALS) cases result from inherited genetic mutations, with about 20% of them attributed to mutations in the gene encoding the ubiquitous cytoplasmic copper/zinc superoxide dismutase 1 (SOD1).
Insilico Medicine Cayman Topco has obtained IND approval from the FDA for ISM-8969 for the treatment of Parkinson’s disease, enabling initiation of a phase I trial in healthy volunteers.
Shionogi & Co. Ltd. and Fosun Pharmaceutical (Group) Co. Ltd. were involved in separate divestment transactions this week, with Shionogi buying out Pfizer Inc.’s stake in Viiv Healthcare Ltd. to lift its holding to 21.7%.
Researchers have developed an AI-based wearable device, called Revoice, to help individuals communicate effectively after a stroke. The system, worn around the neck, uses sensors to capture carotid pulse signals and subtle vibrations from throat muscles to reconstruct intended words and sentences in real time, potentially transforming the lives of stroke patients living with dysarthria.
Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant skeletal muscle disorder with a prevalence of approximately 1 in 8,000. The disorder is driven by aberrant expression of double homeodomain protein 4 (DUX4) within the D4Z4 macrosatellite array. Currently, effective treatments for FSHD are lacking. Strategies aimed at reducing DUX4 expression could hold promise as potential therapeutic approaches for FSHD.
Chengdu Kanghong Pharmaceutical Co. Ltd. has disclosed leucine-rich repeat kinase 2 (LRRK2; dardarin) inhibitors reported to be useful for the treatment of cancer, type 1 diabetes, glaucoma, inflammatory bowel disease, multiple sclerosis, neurodegeneration, psoriasis and rheumatoid arthritis, among others.
Lexicon Pharmaceuticals Inc. announced that it received no objections from the U.S. FDA to move pilavapadin, its non-opioid candidate for diabetic peripheral neuropathic pain, into phase III study, pushing company shares up near 20% on the day.
A team of international researchers is investigating whether a finger-prick blood test could be used to help diagnose Alzheimer’s disease before symptoms appear. With current diagnostic methods relying on expensive brain scans and invasive lumbar punctures, the trial is exploring whether the simple test could offer a faster, cheaper, and more accessible route to identifying the disease.
The good news is that the U.S. Congress is on track to pass a slate of fiscal 2026 spending bills before the current continuing resolution expires Jan. 30. So, barring any last-minute disputes or legislative hostage-taking, there should be no repeat of last year’s 43-day shutdown that impacted NIH grants and activities.
Nippon Zoki Pharmaceutical Co. Ltd. has discovered alkyne derivatives acting as GABA-A receptor subunit α1β2γ2 positive allosteric modulators. As such, they are believed to be useful for the treatment of sleep disorders.
RSS
