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 PMID: https://pubmed.ncbi.nlm.nih.gov/41882406/ (opens in a new tab)
