Pediatric Disease Archives - Xtalks

Wednesday, August 12, 2026 | 11am EDT: Accelerate Cell Culture Media Development with Automated Cell Analysis

Clinical Trial Design in Wolfram Syndrome: Lessons from HELIOS and Rare Disease Research

Why Rare Disease Trial Design Requires the “Right Patients at the Right Time”

FDA Expands Casgevy Approval for Children as Young as 2 With Sickle Cell Disease

Airway Therapeutics CEO on Rethinking Bronchopulmonary Dysplasia Trials

What It Takes to Run Rare Pediatric Disease Trials Across Borders: Insights from Polaryx Therapeutics

International Childhood Cancer Day 2026: Advances in Pediatric Oncology Research and Care

February 2, 2026

Elevidys 3-Year Trial Data Show Sustained Outcomes in DMD Patients

January 30, 2026

New Ultra-Long-Acting Biologic, Exdensur (Depemokimab), Approved for Asthma

December 22, 2025

Diagnostics News

HHS Expands Newborn Screening to Include Duchenne and MLD

December 19, 2025

Waskyra Wins FDA Approval as First Gene Therapy for Wiskott-Aldrich Syndrome

December 12, 2025

Kygevvi Becomes First FDA-Approved Treatment for Rare Mitochondrial Disorder TK2d

November 4, 2025

Modeyso (Dordaviprone) Wins FDA Nod for Rare Brain Tumor Affecting Children and Young Adults

FDA Approves Gamifant (Emapalumab), First Therapy for Macrophage Activation Syndrome in Still’s Disease

Sarepta’s Gene Therapy Elevidys Under Scrutiny After Patient Death

Pregnant participants in clinical trials

January 17, 2025

GUARDIAN Study Reveals How Genomic Sequencing Could Bridge Gaps in Newborn Screening

October 29, 2024

Next Page »