This discovery, linking anaemia in expectant mothers to congenital heart disease (CHD), marks a major step forward in understanding and potentially preventing this common birth defect. Congenital ...

Patients in the EU with Fanconi anaemia (FA) could soon have the first gene therapy option for the disorder, as the EMA starts a review of Rocket Pharma’s RP-L102 candidate. FA is an inherited ...

as well as in anaemia failing an erythropoiesis stimulating agent that requires two or more RBC units over eight week in adult patients with IPSS-R myelodyplastic syndrome with ring sideroblasts ...

In another example of the effect of modifier genes ... 750–1500 mg divided TID-QID Sideroblastic anemia Same free Cu as above Adults and children: 15 mg/kg/d Children: 20 mg/kg/d divided ...

Nat Clin Pract Neurol. 2006;2(9):482-493.

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