Fondazione Telethon brings WASKYRA to the US market, establishing a new standard of care for Wiskott-Aldrich Syndrome patients as the organization scales its clinical distribution network.
The organization is highlighted as the developer of WASKYRA, the first US-approved gene therapy for Wiskott-Aldrich Syndrome, marking a significant commercial milestone.
Fondazione Telethon has officially launched WASKYRA in the United States, marking the first FDA-approved gene therapy for Wiskott-Aldrich Syndrome. The organization is currently expanding its commercial footprint with plans to increase the number of treatment centers in the coming months.
WASKYRA represents the first gene therapy approved in the US for Wiskott-Aldrich Syndrome, providing a critical new treatment option for patients.