Related
NICE Recommends PTC’s AADC Gene Therapy
Rare Daily Staff The United Kingdom’s National Institute for Health and Care Excellence, the drug price watchdog, […]
Read more2023 RARE Mental Health Grant Recipients Announced
Global Genes is pleased to announce the Rare Disease Patient Advocacy Organizations receiving 2023 RARE Mental Health […]
Read moreSeeking a Sustainable Business Developing on N-of-1 Therapies
The ability to pinpoint the underlying genetic causes of diseases and rapidly generate genetic medicines to address […]
Read moreGlobal Genes Team Shares Their Stories on World Rare Disease Day
My daughter Juno was diagnosed with a very rare, severe neurodevelopmental condition called STXBP1-related disorder six years […]
Read moreRARE Global Advocacy Alliance Member Spotlight: Geoffrey Gee
Read moreFinding Me Through This Rare Journey
Patient or Advocate: Brittany Anderson Disease Affiliation: Fibrous Dysplasia, McCune Albright Syndrome, Panhypopituitarism, Adrenal Insufficiency, Trigeminal Neuralgia, […]
Read moreBorn to be rare, born to fight for tomorrow
Patient or Advocate: Nick Irwin Disease Affiliation: Porencephaly Hi. My name is Nick. I’m 6 foot 3 […]
Read moreWhat I Learned About Intrahepatic Cholestasis With Both of My Pregnancies
I frequently wake up thinking of how lucky we were. Before my first pregnancy, I have never […]
Read more