Iron overload was associated with an increased risk for fractures, especially hip, vertebral, and humerus fractures among men and women.

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

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

I am 89 and in good health. For six months, my systolic blood pressure was 113 mm Hg. Then, 10 days ago, it dropped to 88 mm Hg ...

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.

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

Agios Pharmaceuticals, Inc. (AGIO), on Wednesday, announced that its supplemental New Drug Application seeking approval for the ...

About four years after launch, Orna Therapeutics Inc. signed its second major deal, this time validating the lipid nanoparticle delivery technology it acquired through its Renagade Therapeutics Inc.

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

Vertex Pharmaceuticals Inc. will work with a Watertown biotech on new gene-editing therapies for people with sickle cell disease and transfusion-dependent beta thalassemia.

Vertex is paying Orna $65 million upfront, in part via convertible note, according to a Jan. 7 release. Orna—which acquired ...

I am Greek and have thalassemia minor. In general, my red blood cells are much smaller than typical ones, and my iron content ...