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Mar 21, 2026
Research from Germany and Australia shows zebrafish functional testing can identify false-positive SMA newborn screening results, preventing unnecessary treatment in rare cases where infants carry functional SMN1 gene variants.
Mar 04, 2026
Scholar Rock plans to resubmit its biologics license application for apitegromab in spinal muscular atrophy following FDA reinspection of the Catalent Indiana fill-finish facility, with a U.S. launch anticipated in 2026.
Mar 01, 2026
Artificial intelligence is revolutionizing rare disease diagnosis, cutting diagnostic timelines from years to weeks. Rare Disease Day on February 28 highlights challenges faced by over 300 million people worldwide living with more than 7,000 distinct rare conditions.
Feb 28, 2026
International Rare Disease Day on February 28 underscores challenges in diagnosing and treating rare genetic conditions, with patients facing years-long diagnostic delays and geographic barriers to accessing gene therapies.
Feb 27, 2026
Pharmaceutical companies are increasingly partnering with biotechs developing RNA-targeting small molecules, driven by advances in RNA structural biology and the success of drugs like Roche's Evrysdi. The approach aims to address "undruggable" targets while offering oral availability advantages.
Feb 20, 2026
Drug discovery is expanding beyond traditional protein targets to include RNA-targeting small molecules, cell surface proteins, and sequence-based AI platforms capable of screening billions of candidates across the entire genome.
Feb 20, 2026
Former Novartis executive John Tsai has been appointed global head of R&D at Daiichi Sankyo, replacing Ken Takeshita effective April 1. Tsai brings over 25 years of leadership experience in drug development.
Dec 30, 2025
Drug Hunter's most-accessed resources, reviews, and case studies in 2025 reflected key trends in drug discovery, including the rise of oral macrocycles, advances in new modalities, and the shift from biologics to small molecules in immunology.
