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The phosphorylation level of CRMP2 is significantly higher than in controls, severely inhibiting the regeneration capacity of neuronal axons (Arimura et al., 2005)

& Dingemanse, J

These preclinical studies in a -thalassemic mouse model generated the rationale for a phase II proof-of-concept study (ClinicalTrials.gov NCT03692052) of mitapivat in patients with nontransfusion-dependent thalassemias (NTDTs), including both - and -thalassemia genotypes