Minke A E Rab

One-year safety and efficacy of mitapivat in sickle cell disease: follow-up results of a phase 2, open-label study

M. Bartels, B. Biemond, M. Cnossen, 2023, Blood advances.

Biallelic hexokinase 1 (HK1) variants causative of non‐spherocytic haemolytic anaemia: A case series with emphasis on the HK1 promoter variant and literature review

R. van Wijk, B. V. van Oirschot, S. Kakko, 2024, British journal of haematology.

Osmotic gradient ektacytometry – a novel diagnostic approach for neuroacanthocytosis syndromes

K. Peikert, J. Bos, Jonathan R A de Wilde, 2024, Frontiers in neuroscience.

Designing a single-arm phase 2 clinical trial of mitapivat for adult patients with erythrocyte membranopathies (SATISFY): a framework for interventional trials in rare anaemias – pilot study protocol

E. V. van Beers, A. Glenthøj, Fatiha Chermat, 2024, BMJ open.