preheader NTVH

Podcast met prof. dr. Bart Biemond over de meest interessante presentaties over bèta-thalassemie en sikkelcelziekte



  1. ABSTRACT LB2367 | Efficacy and safety of a single dose of CTX001 for transfusion-dependent betathalassemia and severe sickle cell disease.
  2. 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.
  3. 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.
  4. 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.

Klik hier om alleen de audio van deze podcast te beluisteren of te downloaden

Back to previous page

Naar boven