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 ...
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.
Groundbreaking drug treats rare genetic disorder in the womb for first time ever
A two-and-a-half-year-old girl is defying the odds after becoming the first person to be successfully treated for a rare, often fatal genetic condition while still in the womb. The child was conceived ...
This rare genetic disorder was just treated in the womb for the first time ever
This rare genetic disorder was just treated in the womb for the first time ever - Without treatment, those with the more ...
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 ...
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 ...