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rare diseases
Pharma
PTC wins FDA approval for brain-delivered gene therapy Kebilidi
The gene therapy has already been approved to treat AADC deficiency in Europe and the U.K., among other countries, where it's branded as Upstaza.
Zoey Becker
Nov 14, 2024 11:31am
FDA shoots down Intercept's bid for full approval of Ocaliva
Nov 12, 2024 2:12pm
Biologics gaining ground in HS, but more support needed: Spherix
Nov 11, 2024 11:52am
Arrowhead targets rare disease patients with awareness campaign
Nov 5, 2024 11:58am
Novartis, Apellis roll out rival datasets in rare kidney disease
Oct 28, 2024 10:03am
Savara backs talk show episode to boost rare disease awareness
Oct 22, 2024 3:25pm
