Tuberous sclerosis, also called tuberous sclerosis complex (TSC), is a rare genetic multisystem disorder characterized by the ...

Agios Pharma submitted a sNDA to the FDA after two Phase III trials studying PYRUKYND (mitapivat) in thalassemia met their ...

Asia Pacific Leads Next Wave of Sickle Cell Screening Adoption as Public Health Funding Expands Global Sickle Cell Anemia ...