News about "First gene therapy"

US FDA OKs First Gene Therapy for Sanfilippo Syndrome A

US FDA OKs First Gene Therapy for Sanfilippo Syndrome A

The US FDA has approved the first gene therapy for pediatric patients with Sanfilippo Syndrome Type A, a rare and progressive neurodevelopmental disease.

First Gene Therapy | 19/09/2026 | By News Bureau


 

 

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