Neurocrine Lands FDA Nod for First New Treatment in Decades for Rare Endocrine Disorder
A rare, inherited endocrine disorder whose standard treatments are steroids that introduce a wide range of side effects now has ...
Tag: Rare
Texas Woman Who Briefly Died After Rare Delivery Room Complication Has No Memory of Giving Birth to Triplets
A Texas woman retained no memory of giving birth to triplets, including the 48 to 72 hours leading up to ...
Astellas Pharma Lands a First-in-Class Drug Approval in Rare Gastrointestinal Cancer
An Astellas Pharma drug designed to address a novel target has landed a highly anticipated FDA approval for a rare ...
Gene editing approach helps launch first-in-human clinical trial for rare genetic disease
Credit: Unsplash/CC0 Public Domain A collaborative effort between investigators at the National Institutes of Health's National Institute of Allergy and ...
STAT readers on the Rare Disease Innovation Hub and more
First Opinion is STAT’s platform for interesting, illuminating, and provocative articles about the life sciences writ large, written by biotech ...
Sanofi Drug for Rare Blood Disease Is Heading to Recordati for $825M
Sanofi’s Enjaymo, the only approved drug therapy for a rare and debilitating blood disorder, is being acquired by Recordati in ...
TxGNN improves drug repurposing by predicting treatments for rare diseases with no approved therapies
Researchers have developed TxGNN, an AI-powered model that outperforms existing methods by predicting treatments for diseases lacking approved therapies, using ...
How do rare genetic variants affect health? AI provides more accurate predictions
Credit: Pixabay/CC0 Public Domain Whether we are predisposed to particular diseases depends to a large extent on the countless variants ...
US patient dies from rare mosquito-borne disease
Health officials at Sarasota County Mosquito Management Services study specimens of anopheles mosquitoes that cause malaria, in Sarasota, Florida. A ...
Rare disease therapies more feasible, but access remains limited
Will it work? It was the question everyone around Susannah Rosen kept asking. Since infancy, she’d lived with a rare ...
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