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Morning! At the moment, we speak about a JAMA research that outlines the downstream results of the NIH slicing analysis prices, see one man survive a probable case of ALS to date, maybe because of an FUS-targeting drug, and extra.
The necessity-to-know this morning
- Celcuity mentioned its experimental most cancers drug, gedatolisib, delayed tumor development in ladies with a sure kind of breast most cancers, reaching the first targets of a Part 3 scientific trial.
- GSK mentioned it could pay $500 million upfront for rights to an experimental COPD drug from Jiangsu Hengrui Prescription drugs, the most recent instance of a pharma firm turning to a Chinese language agency to construct out its pipeline.
Did this therapy forestall ALS for one man?
After shedding a number of kin to ALS, molecular biologist Jeff Vierstra volunteered for an experimental remedy concentrating on the FUS gene earlier than exhibiting any signs — turning into the primary identified try to stop the deadly illness. Whereas his sisters, who additionally carried the gene and have been additionally handled, each died from falls in 2023 and 2024, Vierstra stays symptom-free practically 5 years later — elevating hope amongst researchers and gene carriers alike for presymptomatic intervention.
