haeomphila A

News

The FDA has granted Breakthrough Therapy Designation to BioMarin’s gene therapy for haemophilia A, helping to intensify competition in the field.

News

Ultragenyx secured its first gene therapy approval, the FDA clearing GENGLYCOS as the first treatment designed to address underlying cause of GSDla.

Digital

A new webinar from Konovo will address some of the critical topics impacting today’s healthcare market research industry.