Good News Network

Successful World First: Baby Treated with Personalized CRISPR Gene Therapy for Rare Disease is Now ‘Thriving’

Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunru crafted a bespoke treatment that has successfully corrected the genetic ...
JD Supra

Will Congress ‘Give Kids A Chance’ by Resurrecting FDA’s Rare Pediatric PRV Program?

An expiring FDA program designed to incentivize research in pediatric rare disease may see new life. The Pediatric Rare Disease Priority ...
NJBIZ

RWJUH named National Rare Disease Center of Excellence (updated)

Rutgers Cancer Institute, together with RWJBarnabas Health, is the state’s only Comprehensive Cancer Center, designated by the National Cancer Institute. The program emphasizes care and research ...

GeneDx CEO Katherine Stueland talks shaking up rare disease diagnosis

GeneDx, a fast-growing biotech doing exome and genomic testing, has seen big returns on Wall Street as appetite for its tests ...
STAT

Can variant interpretation at scale unlock faster answers for rare disease patients?

For patients facing a rare disease, a genetic test isn’t the end of their story; it’s often just the beginning. A test result may point to a diagnosis, or it may reveal a genetic variant that we have ...
Deccan Chronicle

India’s First Rare Disease Hackathon Brings Hope

Global experts at India’s first Rare Disease Hackathon in Hyderabad: Shirlene Badger (UK), Gareth Baynam (Australia), David ...