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News about "rare disease therapy"
Pharming Secures EU Approval for Joenja as First Targeted Treatment for Rare Immune Disorder APDS
Pharming Group has received European Commission approval for Joenja, expanding treatment access for patients with activated PI3K delta syndrome and strengthening therapeutic options for rare immune disorders.
Rare Disease Therapy | 26/05/2026 | By News Bureau
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 | 131
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 | 160
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