Our research strategy

We exist to enable research into aplastic anaemia and other rare bone marrow failures. We encourage and support research that is truly transformative and can accelerate access to better treatments and cures by patients.

Guided by our Patient Support Group and governed by our peer-led Research and Clinical Advisory Panel our research strategy, launched in 2018, focuses on both types of aplastic anaemia - acquired and inherited, in adult and child populations. While the primary focus is on aplastic anaemia, recognising the links with other rare bone marrow failures, we will consider research into dyskeratosis congenita, fanconi anaemia, PNH and others.

Our research priorities

Our research priorities are, to:

  • improve the understanding of rare bone marrow failures in child and adult populations
  • improve the understanding of the diseases by molecular/ genetic research into the damaged cells and their environment
  • identify risk factors in aplastic anaemia patients that may later lead to MDS (Myelodysplastic Syndrome) or AML (Acute Myeloid Leukaemia)
  • improve treatments, identifying alternative cures, enabling experimental therapies and ultimately eradicate the disease