Researchclopedia

Research Researchers Institutions Topics Submit About

Command Palette

Search for a command to run...

Researchclopedia

Research Researchers Institutions About

© 2026 Researchclopedia. Powered by OpenAlex under CC0 license.

Back to research

A blueprint to accelerate rare pediatric gene therapy approvals

20260 citationsJournal Article

Authors

Mohamed Abou-el-Enein · University of Southern California

Mark Walters · UCSF Benioff Children's Hospital

Darshak Sanghavi · University of Massachusetts Chan Medical School

Donald B. Kohn · University of California, Los Angeles

Topics & Keywords

Genomics and Rare Diseases Neurogenetic and Muscular Disorders Research Virus-based gene therapy research

Publication Details

Published in: Nature Medicine

DOI: 10.1038/s41591-025-04115-6

Field-Weighted Citation Impact: 0.00

A blueprint to accelerate rare pediatric gene therapy approvals | Researchclopedia