Wiskott-aldrich Syndrome
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Sign up freeTop drugs studied for this disease
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EMA approval · 2026-03-05EMA Authorised: Waskyra (genetically modified autologous CD34+ cell enriched population that contains haematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human Wiskott-Aldrich Syndrome (WAS) gene) — Fondazione Telethon ETS
EMA authorised Waskyra, a gene therapy using autologous CD34+ cells modified with a lentiviral WAS gene, for Wiskott-Aldrich Syndrome.
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EMA approval · 2025-11-14First gene therapy to treat rare disease Wiskott-Aldrich syndrome
The EMA has approved the first gene therapy for Wiskott-Aldrich syndrome (Waskyra), which significantly reduces infections and bleeding epis…