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Agios Awaits Pyrukynd Decision in Thalassemia as FDA Misses Due Date

AGIO faces uncertainty as the FDA misses its Dec. 7 deadline related to the sNDA for Pyrukynd in thalassemia and offers no new timeline.
Precision Medicine Online

Saol Therapeutics Presses FDA to Reconsider PDCD Drug Without Requiring Another Clinical Trial

The firm will share updated data and new analyses from its Phase III trial with the agency, which issued a complete response ...
BioPharma Dive

FDA raises CAR-T approval standards; Novartis bets on an AI startup

CAR-T cancer therapies now need to succeed in randomized, controlled trials to earn approvals. Elsewhere, a drug from Bristol ...
BMJ Case Reports

Non-type 1 and non-type 2 diabetes in a young man due to novel mutation in HNF1A

Maturity-onset diabetes of the young (MODY) is an autosomal dominant monogenic form of diabetes. This report describes a case with hepatocyte nuclear factor 1-alpha ( HNF1A )-MODY due to a novel ...