News

Fulcrum Therapeutics is advancing its oral therapy, pociredir, for sickle cell disease (SCD) in the PIONEER #trial (NCT05169580). The study is testing safety, effectiveness, and how the drug works in …

Agios Pharmaceuticals is on track to deliver a major milestone in rare disease therapy, with the U.S. FDA decision for PYRUKYND® (mitapivat) in thalassemia expected by September 7, 2025. If …

Vascarta Inc. has initiated a Phase 1 clinical study for VAS-101, a transdermal curcumin gel designed to provide non-opioid pain relief for sickle cell disease (SCD). Conducted by the Foundation …

Emory University researchers have confirmed that hydroxyurea, a widely used oral chemotherapy drug, remains a safe and effective long-term treatment for children with sickle cell disease (SCD). A study tracking …

AB Science has received a U.S. patent for masitinib in treating sickle cell disease (SCD), with protection extending to 2040. The approval follows compelling preclinical results showing masitinib’s efficacy against …

New long-term data from the BELIEVE trial confirm that luspatercept, manufactured by Bristol Myers Squibb, provides lasting benefits for patients with transfusion-dependent beta-thalassemia, significantly reducing the need for frequent blood …

Actinium Pharmaceuticals, Inc. is making progress with Iomab-ACT, an experimental treatment designed to improve stem cell transplants for sickle cell disease (SCD). The therapy uses targeted radiation to clear diseased …

Bristol Myers Squibb receives European Commission approval for Opdivo® + Yervoy® as a first-line treatment for adult patients with unresectable or advanced hepatocellular carcinoma. The Phase 3 CheckMate -9DW trial …

Agios Pharmaceuticals is progressing with its Phase 3 RISE UP trial for Pyrocaine (mitapivat) in sickle cell disease, with results expected by end of 2025. The study aims to demonstrate …