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Waldenström’s macroglobulinemia remains to date an incurable, orphan disease
Given the rarity of WM and its indolent course, which requires long follow-up, robust clinical trial data has been limited and few randomized clinical trials have been conducted to date.
Understanding disease biology and the genomic landscape of the disease are key to the development of new, active and safe agents and predicting response depth and kinetics for each patient.
More than 90% of patients harbor a mutation in MYD88 gene, leading to the constitutive activation of downstream pathways, involving BTK-mediated signaling .
The treatment landscape has been changed with the introduction of BTK inhibitors and ibrutinib (the only EMA/FDA approved drug for the disease), the first-in-class drug has changed paved the way for new tr…