In a major move, the European Medicines Agency (EMA) announced today it is revoking or revising most product class waivers that allow companies to bypass the requirement to test new drugs in pediatric ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today announced that the U.S.

Models predicting pediatric clinician shortages and geographic maldistribution have raised concerns regarding pediatric access to care. The committee will examine clinical workforce trends related to ...

The Digital Medicine Society (DiME) has launched a new set of digital clinical measures to accelerate the development of pediatric rare disease treatments in collaboration with partners including the ...