The Consolidated Appropriations Act of 2026, signed by Trump on Feb. 3, includes a provision reinstating the Mikaela Naylon ...
U.S. President Donald Trump signed a spending package into law Tuesday that reauthorizes the FDA’s previously stalled rare ...
The author's son has hemophilia, a rare disorder requiring expensive, life-saving medication. The TrumpRx program aims to ...
Pharmaceutical Technology on MSN
Rare paediatric disease voucher programme makes long-awaited US return
After more than a year out in the cold, the programme’s reauthorisation is a boost for rare disease therapy developers.
At Web Summit Qatar, AI-powered biotech startups describe how automation, data, and gene editing are filling labor gaps in ...
With a shared purpose, we can realize the true promise of CRISPR and improve healthcare, providing hope to more patients with ...
Investor enthusiasm and evolving FDA pathways are accelerating rare disease drug development, with ultrarare conditions like ...
9don MSN
This 11-year-old has battled a rare disease for years — a breakthrough drug helped deliver relief
Actor turned rare disease advocate Luke Rosen tells how his daughter, Susannah, lives with a KIF1A-associated neurological ...
Hackathons using AlphaGenome and other AI models are hunting down the genetic causes of devastating conditions that have ...
The Orphan Drug Act revolutionized rare disease treatment, yet rising costs and access issues challenge equitable health care solutions.
When you have a rare disease, managing doctor's appointments, prescriptions and care plans is difficult enough — figuring out what Medicare will (and won't) cover often adds another layer of stress.
The seven-year medical odyssey that once defined rare disease diagnosis is being rewritten by algorithms capable of identifying medical zebras in the time it takes to refresh your social media feed.
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