Pediatric Rare 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

The Lily Foundation’s Strategy to Transform Mitochondrial Disease Research

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

Sanofi’s Cablivi Becomes First FDA-Approved Treatment for Pediatric aTTP

January 9, 2026

Forzinity (Elamipretide) Approved by FDA: A First for Barth Syndrome and Mitochondria-Targeted Therapy

October 10, 2025

Sephience (Sepiapterin) for PKU in Children and Adults: New BH4 Precursor Wins FDA Nod

Enhancing Rare Disease Clinical Research Through Patient Advocacy

Sarepta’s Gene Therapy Elevidys Under Scrutiny After Patient Death

On Rare Disease Day 2025, We Need More Than You Can Imagine

February 20, 2025

FDA Approves Gomekli as First NF1 Treatment for Adults & Children

February 13, 2025

Pregnant participants in clinical trials

January 17, 2025

Gene Therapy Skysona Linked to 7 Cases of Blood Cancer

October 15, 2024

Patient-Centered Innovation for Rare Seizure Disorders: Insights from Marinus Pharmaceuticals

September 24, 2024

Clinical Trial News

FDA’s New Rare Disease Innovation Hub to Elevate Patient Care

Lumos Pharma’s Commitment to Innovation in Rare Pediatric Growth Disorders

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