NIMS Medical Genetics department in Hyderabad offers cutting-edge diagnostic and therapeutic services for rare genetic ...

A Pinellas County mom is pushing for more accessible medical food for her 4-year-old son diagnosed with a rare metabolic ...

BioMarin's gene therapy pipeline has had setbacks, including a three-year delay in US approval of Roctavian and stalled development of PKU gene therapy BMN 307 as the Food and Drug Administration ...

Strong Execution and Operational Transformation in 2024 Delivered Record Full Year Results and Provides Momentum for Double-Digit Revenue and Prof ...

Reports Q4 revenue $747M, consensus $711.05M. “Our operational transformation and strong financial execution in 2024 is the first step in ...

SEMINOLE, Fla. (WFLA) — A Pinellas County mother is in a desperate fight to keep her 4-year-old son healthy. Gideon Bennett ...

SEMINOLE, Fla. (WFLA) — A Pinellas County mother is in a desperate fight to keep her 4-year-old son healthy. Gideon Bennett ...

A Seminole mom is fighting for her son's health after a rare disorder left him dependent on a strict medical diet. But now, ...

The biochemist introduced the first heel-prick tests for newborn babies in Spain, protecting infants from life-changing ...

There’s also MZE782, an oral SCL6A19 inhibitor in a phase 1 trial with healthy volunteers and plans to pursue the drug in chronic kidney disease and phenylketonuria. From these raises ...