This report was first published by Endpoints News. To see the original version, click here
The FDA on Tuesday approved a gene therapy for a rare immune disease called Wiskott-Aldrich syndrome, for which patients have little options besides a bone marrow transplant.
The gene therapy will be marketed as Waskyra, and was developed by Fondazione Telethon, an Italian charity focused on rare disease research. It marks the first approved cell and gene therapy from a nonprofit applicant, according to FDA.
您已阅读21%（500字），剩余79%（1828字）包含更多重要信息，订阅以继续探索完整内容，并享受更多专属服务。