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: Janet Woodcock, former FDA official, joins board of patient group focused on ‘rediscovering’ old drugs
STAT Plus: Scholar Rock drug for spinal muscular atrophy succeeds in late-stage study
Sammy Basso, progeria advocate, has died at 28
STAT Plus: Why Biohaven will test the FDA’s regulatory limits
STAT Plus: FDA approves first drug for rare neuron disease, Niemann-Pick type C
STAT Plus: House panel passes pediatric rare disease bill, setting it up for government funding bill
FDA is taking on tampons and ‘misleading’ ads starring Serena Williams
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