rare diseases
News on rare diseases, including the development of new treatments, the challenges of diagnosis and treatment and the stories of patients and their families. The page features in-depth reporting on genetic diseases, immunodeficiencies, metabolic disorders and more.
Latest
STAT Plus: FDA approves first gene therapy for fatal neuron disease in children
STAT Plus: AstraZeneca picks up rare disease-focused Amolyt Pharma in $1 billion deal
STAT Plus: He’s 19 months old and has one of the world’s rarest diseases. His parents are determined to find a cure.
STAT Plus: The FDA isn’t afraid to yank a disappointing drug
Texas Medicaid agrees to fully cover gene therapy for Afghan refugees’ infant
STAT Plus: Obscure vertigo drug could bring rare hope for patients with devastating neuron disease
In partial reversal, Texas Medicaid greenlights first step in gene therapy for Afghan refugees’ infant son
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