Wiskott-Aldrich syndrome is a rare genetic condition that affects about 1 in 100,000 babies, almost exclusively males. It’s caused by mutations in the WAS gene. The syndrome is characterized by 3 main ...

Fondazione Telethon announces the positive opinion issued by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommending marketing authorization in ...

Researchers at the Salk Institute and King Abdullah University of Science and Technology (KAUST) in Saudi Arabia have discovered a new underlying cause of Wiskott-Aldrich syndrome, a rare genetic ...

The U.S. Food and Drug Administration has approved Waskyra (etuvetidigene autotemcel) as the first cell-based gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS). Waskyra is indicated for ...

The rate of severe infections decreased from 2 infections to 0.2 infections per PYO after treatment with etuvetidigene autotemcel. The Food and Drug Administration (FDA) has approved Waskyra ...

Wiskott-Aldrich syndrome is a rare X-linked immunodeficiency characterised by microthrombocytopaenia, eczema and recurrent infections, caused by mutations in the gene encoding Wiskott–Aldrich syndrome ...

Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), announced today that its lentiviral based gene therapy, developed in ...

Researchers reported promising preliminary outcomes for the first four children enrolled in a US gene therapy trial for Wiskott-Aldrich syndrome (WAS), a life-threatening genetic blood and immune ...

Noah Ansari was just 3-months-old when he was diagnosed with a rare and life-threatening immunodeficiency disease called Wiskott-Aldrich syndrome Joelle Goldstein is a Senior News Editor on the TV ...

Researchers have discovered a new underlying cause of Wiskott-Aldrich syndrome, a rare genetic disease that leads to bleeding and immune deficiencies in babies. Their findings revolve around how cells ...

SEX-linked, hereditary thrombocytopenia is a rarely reported disorder. Only 3 cases have been described. 1–3 More commonly reported is the Wiskott–Aldrich syndrome, 4,5 in which sex-linked, hereditary ...