The majority of rare diseases have a genetic cause. The underlying genetic alteration can be found more and more easily, for example by means of exome sequencing (ES), leading to a molecular genetic diagnosis.
World’s first CRISPR-based gene therapy approved for sickle cell anaemia – Pharmaceutical Technology
Share this article Casgevy has been approved for treating sickle cell disease and transfusion-dependent beta thalassemia patients aged 12 and older in the UK. Image