Rare Diseases– tag –

Strategic Overview: Otsuka’s Global Expansion in CNS and Rare Diseases Otsuka Pharmaceutical has focused heavily on the central nervous system (CNS), leveraging U.S.-centered M&A and partnerships to expand its product pipeline. In re...

Strategic Review: Rare Disease, GI, Vaccines, and Innovation Takeda has transformed itself into a global biopharma leader through acquisitions such as Nycomed in 2011 (expanding GI/respiratory reach in Europe), Shire in 2018 (rare diseas...

Why Pediatric Oncology Requires Tailored Innovation From infancy to adulthood, the human body undergoes approximately two decades of dynamic growth. A newborn weighing 3,000 grams grows tenfold or more. Throughout this period, the endocr...

– 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 ...

🧒 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...

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 ...