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 ...
Nature

How DeepMind's genome AI could help solve rare disease mysteries

Hackathons using AlphaGenome and other AI models are hunting down the genetic causes of devastating conditions that have ...
BioSpace

From Awareness to Acceleration: Rare Disease Drug Development Enters a Pivotal Era

Investor enthusiasm and evolving FDA pathways are accelerating rare disease drug development, with ultrarare conditions like ...
BioSpace

Rare Pediatric Disease Vouchers Reauthorized, PBM Reform Funded in Narrowly Passed Spending Bill

U.S. President Donald Trump signed a spending package into law Tuesday that reauthorizes the FDA’s previously stalled rare ...
PharmTech

Rare Disease Treatments: Navigating the Economics of Global Innovation

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.
Fierce Pharma

FDA’s rare pediatric disease voucher program revived by 2026 government funding bill

The Consolidated Appropriations Act of 2026, signed by Trump on Feb. 3, includes a provision reinstating the Mikaela Naylon ...

Pearl Jam’s Eddie Vedder fights rare disease in NOTL filmmaker’s new documentary

Niagara-on-the-Lake’s Matt Finlin has built an accomplished career in the not-for-profit and film worlds, producing ...

Hot Picks: Biotech rebound turns to rare disease catalysts

Biotech stocks rebounded in 2025. Wedbush’s Laura Chico explains why rare disease catalysts and upcoming data could shape the ...

Rare Michelangelo foot sketch for Sistine Chapel fresco fetches record €23 million at auction

Study for a foot of the Libyan Sibyl/Michelangelo (1475-1564) ...

A rare disease took her sister. Now, a Burleson mom is helping search for a cure

Alström syndrome affects roughly one in 500,000 people. In May, Fort Worth will host a symposium of researchers looking into treatments.

South Carolina kids are developing rare brain swelling after measles

Children in South Carolina are developing rare brain swelling complications after measles outbreak - The majority of the ...