Agios Pharma submitted a sNDA to the FDA after two Phase III trials studying PYRUKYND (mitapivat) in thalassemia met their ...

Agios Pharmaceuticals AGIO announced that the FDA has accepted its supplemental new drug application (sNDA) seeking label ...

A phase 3 trial highlights betibeglogene autotemcel as a potentially curative gene therapy for severe transfusion-dependent β ...

Mumbai: Families of children undergoing treatment at BMC's low-cost thalassemia care and bone marrow transplant centre in ...

It looks like you do not have any active subscriptions. To get one, go to the subscriptions page. DEAR DR. ROACH: I am Greek and have thalassemia minor. In general, my red blood cells are much smaller ...

On the special invitation of Punjab Assembly Speaker Malik Mohammad Ahmad Khan, a delegation of children of Sundas Foundation ...

The sNDA is supported by data from the phase 3 ENERGIZE and ENERGIZE-T studies, which evaluated mitapivat vs placebo in patients with NTD and TD alpha- or beta-thalassemia, respectively.

Achieved in vivo preclinical proof of concept of editing hematopoietic stem cells in non-human primates as a key step toward developing a novel ...

The three-year partnership is another example of Vertex’s interest in improving on Casgevy, its CRISPR medicine for sickle ...

FDA Accepted Agios’ Supplemental New Drug Application for PYRUKYND® (mitapivat) in Adult Patients with Non-Transfusion-Dependent and ...

The camp at Suraj Bhawan in Ghasi Bazaar Market was aimed at providing vital support to individuals suffering from the ...