Life Science Leader4d
A Father's Perspective On The Rare Pediatric Disease PRV Program
The FDA’s Rare Pediatric Disease Priority Review Voucher (PRV) program is set to expire on December 20, 2024, if the U.S.
coloradosun5d
A 10-year-old Parker girl’s disease is one in a million, and advancements in her lifetime made all the difference
Juliette is one of just 33 children in the world taking a new injectable drug to keep her rare blood disease from attacking ...
Collaborative seeks to change children's lives through genetics
In the course of her care, she joined the Children's Rare Disease Collaborative (CRDC), a hospital-wide effort to ...
9-year-old Jordy is flying high after battling rare genetic disease | 2024 Free Care Fund
At age 4, a pediatrician sent Jordy Greene to UPMC Children's Hospital of Pittsburgh because he was going down on the growth ...
Yahoo7d
Rush University Medical Center receives gifts for children with rare diseases
Christmas gifts picked out for children battling rare diseases were dropped off Saturday morning at Rush University ...
Minnesota Daily10d
Parents of children with rare diseases share their stories for Rare Disease Day
The University houses the Rare Disease Advisory Council (RDAC), a support network for parents of children with rare diseases. The University has helped support families in dealing with ...
KSTP-TV11d
Rare disease program needs Congress to act to keep key drugs in development
In the U.S., about 30 million people—half of them children—have a rare disease, according to the U.S. Government ...
He developed ‘puff of smoke’ in his brain from moyamoya disease
It is a rare, progressive cerebrovascular disorder caused by blocked arteries at the base of the brain. Read more at ...
STAT12d
Mandatory milk testing and a beacon in the rare-disease world
Get your daily dose of health and medicine every weekday with STAT’s free newsletter Morning Rounds. Sign up here. Good ...