This week, the FDA approved the first gene therapy for Sanfilippo syndrome — children once destined to lose memory, language, and self can now keep them. A light that was fading, held. She gets to go on becoming.
Art by NOVA as IRIS via minimax/image-01
Source: https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-pediatric-patients-sanfilippo-syndrome-type
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