Opinion

America's Rare Children Need Congress to Act: NORD Urges Swift Reauthorization of Proven Rare Pediatric Disease Voucher Program

The National Organization for Rare Disorders (NORD(R)) today urged Congress to reauthorize the Rare Pediatric Disease Priority Review Voucher (RPD PRV) program by year-end, warning that continued ...
FOX 32 Chicago on MSN

Lurie Children's launches genetics and rare diseases division behind $11M gift

Ann & Robert H. Lurie Children’s Hospital of Chicago has received an $11 million gift to establish the Edwards Family Division of Genetics and Rare Diseases.

First Baby to Undergo Gene Editing Takes First Steps: ‘It’s All Been a Miracle’

The first infant to undergo gene-editing therapy has accomplished another first — his first steps. KJ Muldoon is walking and ...
KWTX

Parents keep faith as their children battle a rare disease, affecting only 30 kids in US

QUINCY, Ill. (WGEM/Gray News) - The Kempf family says they have received devastating news about their children when it comes to a rare disease. Both of their children have been diagnosed with ...
Opinion
MinnPostOpinion

Congress can save lives with Rare Pediatric Disease PRVs

For Americans living with rare diseases, the Rare Pediatric Disease Priority Review Voucher Program is a vital tool. Yet its reauthorization is at risk.
TipRanks on MSN

Amgen’s pediatric inebilizumab study signals long-term upside in rare autoimmune disease

Amgen Inc ($AMGN) announced an update on their ongoing clinical study. Amgen’s latest clinical move targets a niche but ...