Rare & Pediatric Disease– category –
-
Rare & Pediatric Disease News Vol.4: Organoid-Based Discovery for UBA5-Linked Pediatric Epileptic Encephalopathy
Screenshot Human Brain Organoids Reveal Therapeutic Potential for Rare Pediatric Epilepsy In June 2025, a research team led by Dr. Heather Mefford at St. Jude Children’s Research Hospital reported a groundbreaking study using human brain... -
Rare & Pediatric Disease News Vol.2: How Far Can Japan Catch Up in Pediatric Rare Disease Drug Development?
– Global Comparison and Future Perspectives in the Post-PRV Era – With the expiration of the U.S. FDA’s Rare Pediatric Disease Priority Review Voucher (PRV) program in 2024, a global debate has emerged about how to sustain innovation in ... -
Rare & Pediatric Disease News Vol.1
🧒 Introduction: Why Focus on Pediatric Rare Diseases? Pediatric rare diseases often involve life-threatening conditions with few treatment options. In 2012, the U.S. introduced the Rare Pediatric Disease Priority Review Voucher (PRV) pro... -
Rare & Pediatric Disease News Vol.3 : New Gene Therapy Approach for Rare Childhood Blindness: Drug Discovery News Spotlight
A U.S. research team has developed a novel gene therapy for Leber Congenital Amaurosis (LCA), a rare form of childhood blindness. Unlike conventional AAV-based gene augmentation, this approach utilizes RNA editing technology to directly ...
1