The recent findings on consanguinity rates in Sri Lanka by Dr. Thurairajah’s team, as reported in The Sunday Times, have ...

Exagamglogene autotemcel (exa-cel), a CRISPR-Cas9 gene-edited cell therapy, led to sustained improvements in health-related quality of life (HRQOL) for both adults and adolescents with ...

Shares of Agios Pharmaceuticals AGIO fell 11% on Thursday after the company announced that the FDA had extended the review timeline for the supplemental new drug application (sNDA) seeking label ...

PDUFA goal date extended by three months from September 7, 2025, to December 7, 2025 CAMBRIDGE, Mass., Sept. 04, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage ...

The new Prescription Drug User Fee Act target date is now December 7, 2025. The Food and Drug Administration (FDA) has extended the review period for the supplemental New Drug Application (sNDA) for ...

Tempus AI has acquired the digital pathology developer Paige, including its FDA-cleared, artificial-intelligence-powered programs for spotting the signs of cancer. The deal totals $81.25 million, ...

An essential factor in distinguishing thalassemia phenotypes is transfusion dependence. While beta-thalassemia major requires life-long blood transfusions for survival, non-transfusion-dependent ...

CLEVELAND, Ohio -- Cleveland Clinic Children’s Hospital is among the first in the country to use a new gene therapy to treat patients with thalassemia, a genetic blood disorder that affects the ...

China announced Wednesday that four children with thalassemia, a severe genetic blood disorder usually diagnosed in toddlers, have been cured using a domestically developed DNA base editing therapy.

Beta thalassemia is a treatable blood disorder that’s inherited, or passed down through your genes. With beta thalassemia, your body doesn’t make enough hemoglobin, which is an important protein used ...

The Food and Drug Administration (FDA) has accepted for review the supplemental New Drug Application (sNDA) for mitapivat for the treatment of adults with non-transfusion-dependent (NTD) and ...

Treatment effects sustained through long-term follow-up of beyond 10 years in the earliest treated patients (n=2); 81% of participants have >5 years of follow-up SOMERVILLE, Mass.--(BUSINESS ...