Last edition
News about "rare disease therapy"
Johnson & Johnson Secures FDA Priority Review for Imaavy in Rare Blood Disorder
Johnson & Johnson moves closer to delivering a first-in-class treatment for Warm Autoimmune Haemolytic Anaemia with FDA Priority Review for Imaavy.
Rare Disease Therapy | 28/04/2026 | By News Bureau
Thryv Therapeutics Advances THRV-1268 Programme with Phase II/III Trial and FDA Fast Track Status
Thryv Therapeutics initiates Phase II/III clinical testing and secures FDA Fast Track status for THRV-1268, advancing a potential first-in-class therapy for Long QT Syndrome.
Rare Disease Therapy | 27/04/2026 | By News Bureau
Sanofi Secures FDA Breakthrough Designation for Rare Disease Therapy
Sanofi has received Breakthrough Therapy designation from the US Food and Drug Administration (FDA) for its investigational drug venglustat, aimed at treating neurological symptoms associated with type 3 Gaucher disease (GD3).
Rare Disease Therapy | 19/03/2026 | By Darshana | 137
We use our own and third party cookies to produce statistical information and show you personalized advertising by analyzing your browsing, according to our COOKIES POLICY. If you continue visiting our Site, you accept its use.
More information: Privacy Policy