A blueprint to accelerate rare pediatric gene therapy approvals.

A blueprint to accelerate rare pediatric gene therapy approvals.
Abou-El-Enein M, Walters MC, Sanghavi D, Kohn DB. Nat Med. 2026 Apr;32(4):1170-1171. doi: 10.1038/s41591-025-04115-6. PMID: 41882406