6don 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 ...
Highlights advancing clinical-stage pipeline, including two major value-creating programs for primary immunodeficiencies ...
Hackathons using AlphaGenome and other AI models are hunting down the genetic causes of devastating conditions that have ...
GeneDx, a fast-growing biotech doing exome and genomic testing, has seen big returns on Wall Street as appetite for its tests ...
Investor enthusiasm and evolving FDA pathways are accelerating rare disease drug development, with ultrarare conditions like ...
U.S. President Donald Trump signed a spending package into law Tuesday that reauthorizes the FDA’s previously stalled rare ...
Over the past four decades, rare disease drug development has evolved from a scientific aspiration into a central pillar of biopharmaceutical innovation. Regulatory reforms, scientific breakthroughs, ...
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.
The Consolidated Appropriations Act of 2026, signed by Trump on Feb. 3, includes a provision reinstating the Mikaela Naylon ...
Niagara-on-the-Lake’s Matt Finlin has built an accomplished career in the not-for-profit and film worlds, producing ...
World Health Organisation (WHO) director general Tedros Adhanom Ghebreyesus on Saturday warned that cases of Nipah virus are rare but also serious. The global health body head also said that cases ...
Biotech stocks rebounded in 2025. Wedbush’s Laura Chico explains why rare disease catalysts and upcoming data could shape the ...
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