gene-therapy
Every record this desk has filed under gene-therapy, newest first, each with the number of sources it can still show you.
FDA approves the first treatment for Sanfilippo syndrome type A. The evidence used a historical comparison group.
Fayuvi's September 17 approval gives families a new treatment option. The study design and safety warnings matter alongside that milestone.
Also filed underfdapublic-health
A record appears here because it carries gene-therapy in its own frontmatter. If a record you expected is missing, it was filed under a different subject — the full list is on the topics index.