Treating Dyskeratosis Congenita will vary from patient to patient.


1) Treatment is with tablets to improve telomere repair such as danazol.

Increasing Blood Cell Production

2) Injections or tablets to increase blood cell production such as EPO (erythropoietin) injections (for anaemia), GCSF injections (for low neutrophil white blood cells), eltrombopag (for low platelets and sometimes for anaemia).

Bone Marrow or Stem Cell Transplant

3) Bone marrow or stem cell transplantation in very severe cases. Bone marrow/stem cell transplantation should be carried out under the guidance of a specialist familiar with dyskeratosis congenita, as it is important to avoid medications that have side effects such as lung fibrosis or excessive clotting.