In 2024, regulators approved Casgevy, the first CRISPR-based therapy to treat sickle cell disease and beta-thalassemia — a ...

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions. A single one-time gene therapy could free ...

This is read by an automated voice. Please report any issues or inconsistencies here. It’s a shrug. That’s the part that gets you. We ask the question every day, a dozen times a day. “Any bleeding ...

ATLANTA and BOSTON, Sept. 15, 2025 (GLOBE NEWSWIRE) -- ABLi Therapeutics (“ABLi”), a biotechnology company developing therapeutics to address diseases that arise from activation of Abelson Tyrosine ...

Many people have a bleeding disorder and don't know it, but the Western Pennsylvania Bleeding Disorders Foundation is working to help people recognize the signs and get support. The foundation helps ...

CRESTVIEW HILLS, Ky. (WKRC) — The Thomas More University men's soccer team welcomed a special member, Cohen Bramlee, to a game in person for the very first time. Cohen Bramlee was diagnosed with a ...

Children's Minnesota is hosting the fundraiser in September for Childhood Cancer Awareness Month and Sickle Cell Awareness Month. People can donate online and their gift amount will be doubled by the ...

Sanofi’s drug for a rare autoimmune blood disorder won approval in the US, raising the prospect of the medicine becoming a blockbuster. The drug rilzabrutinib, which will go by the brand name Wayrilz, ...

Sanofi received Food and Drug Administration approval for Wayrilz, a treatment for adults with persistent or chronic immune thrombocytopenia who have had insufficient response to a prior treatment.

For Novartis, this week is all about ianalumab. A day after posting a pair of phase 3 wins for the tricky indication of Sjögren's syndrome, the Swiss pharma has unveiled a separate late-stage victory ...

Shares of PTC Therapeutics climbed after the company said federal regulators approved its treatment for elevated levels of a certain amino acid in the blood. The stock was up 11%, at $49.08, midday ...