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 ...
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 ...
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 ...
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 ...
HealthDay on MSN
FDA Approves Waskyra for Wiskott-Aldrich Syndrome
The FDA has approved Waskyra (etuvetidigene autotemcel) as the first cell-based gene therapy for the treatment of WAS.
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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