A Pinellas County mother is in a desperate fight to keep her 4-year-old son healthy. Gideon Bennett has a rare genetic ...

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

Government of India has provided tax relief on life-saving drugs and medicines used for rare diseases. As per the Ministry of Finance’s response in Rajya Sabha on February 11, 2025, basic customs duty ...

A Seminole mom is fighting for her son’s health after a rare disorder left him dependent on a strict medical diet. But now, she’s facing an unexpected challenge: losing access to the ...

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

PTC Therapeutics (PTCT) saw its shares surge in the last session with trading volume being higher than average. The latest ...

PTC Therapeutics, Inc. (NASDAQ:PTCT), a biopharmaceutical company focused on developing and commercializing novel medicines for rare disorders, finds itself at a critical juncture. With a promising ...

PTC Therapeutics (PTCT) is advancing 8% after investment bank Cantor Fitzgerald’ hiked its price target on the drug maker’s ...

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 ...

Genzyme Corp. (Sanofi Genzyme) has disclosed sodium-dependent neutral amino acid transporter B(0)AT1 (SLC6A19) inhibitors reported to be useful for the treatment of phenylketonuria, among others.