ENERGIZE-T Study Achieved Primary and All Key Secondary Endpoints in Adult Patients with Transfusion-Dependent Alpha- or Beta-Thalassemia –– ...

Thalassemia is a group of blood problems, not just one. The two types are: Alpha thalassemia, which happens when one or more genes for the alpha globin protein are missing or mutated Beta ...

The fall in AGIO stock comes after reports of an unexpected side effect linked to its sole-marketed drug Pyrukynd in thalassemia patients.

Vertex Pharmaceuticals (VRTX) presented positive long-term data for its gene therapy Casgevy and issued a business update on ...

Agios Pharmaceuticals (AGIO) presented positive results from the Phase 3 ENERGIZE-T study investigating mitapivat, an oral, small molecule PK ...

Shares of Agios Pharmaceuticals collapsed Monday on an unexpected side effect related to the company's thalassemia treatment, mitapivat.

There are two major types of thalassemia: alpha and beta. Alpha globin and beta globin are the building blocks of hemoglobin. Those with alpha thalassemia don't make enough alpha globin and those with ...

Vertex Pharmaceuticals Incorporated looks set for two new drug approvals in early 2025. Click here to read an analysis of ...

Fintel reports that on December 9, 2024, Jefferies upgraded their outlook for Vertex Pharmaceuticals (NasdaqGS:VRTX) from ...

Hematologists and oncologists from around the world will present new research and clinical findings at the American Society of Hematology’s (ASH) 66th Annual Meeting and Exposition. This year’s ...

Beam Therapeutics (NASDAQ:BEAM – Get Free Report)‘s stock had its “buy” rating reiterated by stock analysts at HC Wainwright ...