First gene therapy to treat rare disease Wiskott-Aldrich syndrome

View source ↗

Summary

The EMA has approved the first gene therapy for Wiskott-Aldrich syndrome (Waskyra), which significantly reduces infections and bleeding episodes in affected ind…

Upgrade for the full analysis, importance score, and drug timeline.

Diseases / indications

• Wiskott-aldrich Syndrome

Drugs mentioned

• Waskyra

Snippet

Waskyra significantly reduces infections and bleeding episodes in people with life-threatening immunodeficiency