Actor turned rare disease advocate Luke Rosen tells how his daughter, Susannah, lives with a KIF1A-associated neurological ...

The Senate failed to pass a massive spending bill on Thursday—which includes the rare pediatric PRV program but also funding for the Immigration and Customs Enforcement’s large-scale crackdown in ...

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

Patient groups argue that customs exemptions for rare disease treatments offer minimal relief without sustained government ...

Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis ...

Budget 2026 exempts drugs for cancer and rare diseases from customs duties, but concerns persist about actual patient ...

Research on rare diagnoses and the development of precision medicine depend on patients being able to share their health data in a secure and ethical manner. The research study, published in ...

Torie Bosch is the First Opinion editor at STAT. Celena Lozano’s son Benny, who turned 5 in November, loves trains, trucks — anything that goes. He also has a rare disease, PURA syndrome. Earlier this ...

A 2-year-old boy died from a rare disease with flu-like symptoms, just weeks before his third birthday. Hudson Hughie Martin, who was diagnosed with the rare Kawasaki disease when he was just 7 months ...

XCells Biotechnologies ("iXCells"), a leading provider of human cell-based solutions and custom iPSC services, and Rosebud ...

Union Budget 2026-27 brings major relief for cancer and rare disease patients by cutting import duties on costly medicines.