Switching On the Silent Gene in Rett Syndrome

• August 25, 2025

The Dynamics of Neurofilament Light Chain in Spinal Muscular Atrophy

• by Arlene D'Silva, Karen Herbert, Lakshmi Balaji, Jia Mei He, Tejaswi Kandula, Hugo A. Sampaio, Hooi-Ling Teoh, Esther Tantsis, Jihee Sohn, Nancy Briggs, Nickson Ning, Matthew C. Kiernan, Didu S. Kariyawasam, Michelle A. Farrar

• July 1, 2026

FDA approves gene therapy for MPS IIIA

The one-time intravenous treatment uses adeno-associated virus serotype 9 to deliver a functional copy of the SGSH gene.

• by Kathryn Wighton

• September 18, 2026

• 2 min

Emergency diagnosis may flag poor outcomes beyond cancer

Across 13 noncancer conditions, emergency diagnosis was common and associated with higher 1-year mortality.

• by Andrea Surnit

• August 31, 2026

• 4 min