Bristol-Myers Squibb Company (($BMY)) announced an update on their ongoing clinical study. Study Overview: Bristol-Myers ...

Marathon's Alpha test is going on right now, and despite NDAs, we know a lot about it, including how much it appeals to ...

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) is one of the best long term low volatility stocks to buy right now.

Patients with sickle cell disease and beta-thalassemia treated with exagamglogene autotemcel reported substantial and ...

PYRUKYND is currently approved in Europe for adults with PK deficiency; thalassemia would represent its second indication, ...

Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced that the Committee ...

Agios Pharmaceuticals said a key European regulatory committee has recommended expanded approval of its Pyrukynd anemia drug for certain people with the inherited blood disorder thalassemia. Agios on ...

CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced the acceptance of an abstract for oral ...

Vertex Pharmaceuticals (Nasdaq: VRTX) announced today a reimbursement agreement with the Italian Medicines Agency (AIFA) for eligible transfusion-dependent beta thalassemia (TDT) and severe sickle ...

Thanks to groundbreaking in-utero blood transfusion technology, what was once a fatal diagnosis in the womb can now result in live births. However, this medical advancement created a new challenge: a ...

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 ...

In the beta thalassemia study, Dr. de la Fuente and his colleagues used two quality of life scales to evaluate outcomes in adult patients and two quality of life scales for adolescent patients. Unlike ...