tag orphan-drug-act 
Pharmaceutical Technology
JUNE 29, 2022
The US Food and Drug Administration (FDA) put a high-profile bluebird bio trial for sickle cell disease on partial clinical hold, and advisory panels deliberated over decisions involving gene therapies for amyotrophic lateral sclerosis (ALS), cerebral adrenoleukodystrophy (CALD), and beta-thalassemia.
pharmaphorum
JUNE 2, 2022
R&D into orphan drugs is growing alongside the number of approved treatments, providing treatments for rare diseases that previously did not have any. 141/2000 (the Orphan Regulation), that both acted to encourage development in the area, are regarded as crucial for changing the landscape in rare disease R&D.
Pharmaceutical Technology
JULY 14, 2022
It is estimated that there are currently more than 7,000 orphan diseases, many of which are considered life-threatening and most of which have a genetic basis. Despite this high number, orphan diseases are rare by definition, affecting around one in 2,000 people as defined by the European Union. Efforts have been made to change this.
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