Philippine Daily Inquirer2d
National Rare Disease Week: Easing the pain of suffering in silence
For many Filipinos, receiving a medical diagnosis is supposed to provide clarity. But for those with rare diseases, it often ...
Rare rearing: Japanese collaboration to save near-extinct White-bellied herons in Bhutan takes wings
The White-bellied Heron (WBH), the world's second-largest heron, is a symbolic bird for the people of Bhutan and also a ...
Model born with rare birth defect uses humor to destigmatize limb differences
Lyric Mariah Heard spoke with "Good Morning America" about her life as a model born with a rare birth defect and using humor ...
Daily Mail on MSN2d
Man was born with a deformity after being exposed to nuclear radiation
There's a Rare Chance to See the Northern Lights Tonight — Here's How to Watch Vikings Predicted To Add $49.6 Million, 377-TD ...
MedPage Today1d
FDA Approves First Drug for Rare Lipid Storage Disorder
Chenodiol -- a naturally occurring human bile acid also known as chenodeoxycholic acid -- is the first FDA-approved drug indicated for the rare lipid storage disease and autosomal recessive genetic ...
This rare genetic disorder was just treated in the womb for the first time ever
Following the first-ever treatment for spinal muscular atrophy in the womb, physicians say a 2-year-old girl shows no signs ...
Advocating for a child with a rare disorder brings heartbreak. And then there’s the joy.
When a child might not be — when something is wrong, yet no one seems to know what it is — the anxiety is soul-crushing. And ...
Autophagy malfunction study moves a step closer to understanding Rett syndrome
Rett syndrome is a rare genetic disease affecting 1 in 10,000 newborn girls, characterized by sudden regression around 1 year ...
US FDA approves Mirum Pharma's genetic disorder drug
The U.S. Food and Drug Administration approved Mirum Pharmaceuticals' genetic disorder drug on Friday, making it the first ...
1MIRM : FDA Approves Mirum Pharmaceuticals' Drug As First For Genetic Disorder...
Mirum Pharmaceuticals' Ctexli wins FDA approval for CTX, a rare lipid storage disease. The company projects 2025 global sales of $420–$435 million.