FDA and CMS Announce New Steps to Advance Gene Therapies and Expand Access for Patients with Rare Diseases

• by Assistant Secretary for Public Affairs (ASPA)

• October 27, 2026

Parental perspectives on gluten immunogenic peptide urine testing in children with coeliac disease: a qualitative study on barriers, emotions and interest in point-of-care tools

• by Aisling Fiuza, Alfonso Rodriguez-Herrera

• October 9, 2026

A Community-Based Behavioral Intervention and Body Mass Index in School-Aged Children: A Cluster Randomized

• by Monica L. Wang, Selenne Alatorre, Aviva G. Schwarz, Janice Weinberg, Linda Sprague Martinez, Jennifer Aldworth, Rabindra Kadel, Milagros C. Rosal, Stephenie C. Lemon

• October 9, 2026

First MCT8 deficiency therapy approved

Clinical studies showed reductions in circulating thyroid hormone levels and changes in heart rate and systolic blood pressure.

• by Kathryn Wighton

• September 29, 2026

• 3 min