Fanconi anaemia (FA) is an autosomal recessive disease that is characterized by developmental abnormalities, cancer susceptibility and cellular sensitivity to crosslinking agents. The seven cloned ...

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

Anemia happens when your blood doesn’t carry enough oxygen to your body — usually because your blood isn’t making enough healthy red blood cells. Although anyone can get anemia, it’s more common in ...

Sickle cell disease is an autosomal recessive blood disorder that can lead to anaemia. It is caused by a mutation in the haemoglobin gene, which leads to deformation of red blood cells. Deformed ...