Podcast met prof. dr. Bart Biemond over de meest interessante presentaties over bèta-thalassemie en sikkelcelziekte
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- POSTER P1453 | Stable transduction of fetal hemoglobin in patients with sickle cell disease in the phase 1/2 MOMENTUM study of ARU-1801 gene therapy and reduced intensity conditioning.
- ABSTRACT S268 | Safety, tolerability, and pharmacokinetic/pharmacodynamics results from phase I studies of GBT021601, a next-generation HBS polymerization inhibitor for treatment of sickle cell disease.
- POSTER P1502 | Long-term follow-up of Dutch patients with sickle cell disease diagnosed by neonatal screening – effect on the morbidity and mortality in the Netherlands.
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