Rare Disease News, Perspectives & Resources - Rare Disease …
WEBRare Disease News is the premier online resource for the latest rare disease news, perspectives and information.
The 10 Biggest Stories in Rare Disease for 2023
WEBDec 13, 2023 · The 10 Biggest Stories in Rare Disease for 2023. From landmark gene therapy approvals to illuminating discoveries into the pathogenesis of rare diseases, 2023 saw substantive advances in rare disease diagnosis and treatment.
Rare Disease News - National Organization for Rare Disorders
WEB4 days ago · Research. The National Organization for Rare Disorders (NORD) and the Rare Disease Diversity Coalition (RDDC) have unveiled the findings of a groundbreaking national survey aimed at illuminating the barriers faced by … Read more. Congress: Reauthorize the Rare Pediatric Disease Priority Review Voucher Program.
Featured News - National Organization for Rare Disorders
WEBAug 27, 2024 · The National Organization for Rare Disorders (NORD) and the Rare Disease Diversity Coalition (RDDC) have unveiled the findings of a groundbreaking national survey aimed at illuminating the barriers faced by …. Read more.
UCL researchers deliver breakthrough head-to-head clinical trial in ...
WEB1 day ago · Published in Lancet Neurology, UCL researchers have delivered a ground- breaking investigator-led head-to-head rare disease trial that will impact clinical practice globally. Approximately 1 in 17 UK people have a rare disease and the …
House panel passes pediatric rare disease bill - STAT
WEB1 day ago · WASHINGTON — A House committee on Wednesday passed a bill to renew a program aimed at aiding development of drugs for rare diseases in children, putting it in a better position to be added to...
New Mayo Clinic technology helps solve the unsolvable in rare disease ...
WEBJun 5, 2024 · Mayo Clinic researchers have developed a semiautomated system, known as RENEW, to rapidly re-analyze unresolved rare disease cases.
National Survey Reveals Impact of COVID-19 on People Living with Rare …
WEBFeb 16, 2024 · RDCRN researchers share insights from a survey about impacts of the COVID-19 pandemic on individuals with rare diseases, families, and caregivers. The devastating impact of COVID-19 on the general population is well-documented—but less is known about the millions of people living with rare diseases.
National Organization for Rare Disorders | NORD
WEBAug 27, 2024 · NORD advances practical, meaningful, and enduring change so people with rare diseases can live their fullest and best lives. Every day, we elevate care, advance research, and drive policy in a purposeful and holistic manner to lift …
Complex trait associations in rare diseases and impacts on
WEB2 days ago · Polygenic scores aggregate the effects of common genetic variants and have been shown to underly certain rare diseases.