Wiskott-Aldrich syndrome is a very rare genetic disorder that affects the WAS gene. Usually seen in males, it’s present from birth and characterized by a specific triad of symptoms. Wiskott-Aldrich ...
The FDA has approved Waskyra (etuvetidigene autotemcel), the first gene therapy for Wiskott-Aldrich syndrome, a rare and life-threatening immune disorder. Waskyra is indicated for pediatric patients 6 ...
DelveInsight’s “Wiskott-Aldrich Syndrome Pipeline Insight 2025” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in the Wiskott-Aldrich Syndrome pipeline landscape. It ...
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 ...
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 ...
WAS affects almost exclusively males, with an estimated incidence of 1 in 250,000 live male births. The therapy represents a major scientific and clinical achievement, offering new hope for patients ...
A genetic disorder that leads to bleeding episodes and life-threatening infections has a new FDA approved treatment, a regulatory decision that marks two notable firsts — the first gene therapy for ...
The US FDA has approved the first ever cell-based gene therapy to treat Wiskott-Aldrich syndrome (WAS). The gene therapy, Waskyra (etuvetidigene), can be used to treat adults with WAS who have a ...
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