RCCS San Raffaele Scientific Institute in Milan researchers have discovered that hematopoietic stem cells (HSCs) adapt their lineage commitment during gene therapy based on the underlying genetic ...

This case study is an example of how the MHRA is benefiting from patients providing their lived experience for the pre-authorisation stage of the ...

Beam Therapeutics enrolls 35 patients in BEACON trial for BEAM-101 in sickle cell disease, with promising early safety and ...

CASGEVY approved for the treatment of patients 12 years of age and older with sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) in Switzerland and Canada- -45 authorized treat ...

With new approvals on the horizon and the closely watched Casgevy launch picking up steam in sickle cell disease and beta ...

CASGEVY™ approved for the treatment of patients 12 years of age and older with sickle cell disease (SCD) and transfusion-dependent beta ...

Results from Phase 3 ENERGIZE-T Study of Mitapivat in Transfusion-dependent Thalassemia will be Presented in Oral Session –– Tebapivat Phase 1 ...

Q3 2024 Earnings Call Transcript October 31, 2024 Operator: Good morning, and welcome to Agios’ Third Quarter 2024 Conference Call. [Operator Instructions] Please be advised that today’s call is being ...

In-depth interviews and an online survey found support for gene therapy as a treatment for inherited blood disorders, but ...

Doctors stress the importance of awareness and testing for thalassemia, a genetic blood disorder affecting children, as cases continue to rise due to lack of information and government programs.

BAKU, Azerbaijan, October 31. Mehriban Aliyeva, First Vice-President of the Republic of Azerbaijan and President of the ...