P037: In vitro evaluation of drug–drug interaction potential of doxecitine and doxribtimine in thymidine kinase 2 deficiency (TK2d)doi:10.1016/j.gimo.2025.102881Aravind MitturUCB, Morrisville, NCPam SantiagoUCB, Emeryville, CAMelanie Golding...
MT1621 is a novel investigational deoxynucleoside substrate enhancement therapy to treat Thymidine Kinase 2 deficiency (TK2d) In February 2019, Modis Therapeutics received Breakthrough Therapy designation from U.S. FDA for MT1621, an investigational deoxynucleoside combination therapy used for the ...
This is a very rare disease and there are few cases described in literature, we describe the respiratory involvement we have observed in a series case.Methods: Observational prospective study of the respiratory evolution of 6 patients with late onset thymidine kinase 2 deficiency (LOTK2D), ...
Thymidine kinase 2 deficiency (TK2d) is an ultra-rare, genetic mitochondrial disease associated with progressive proximal myopathy and respiratory weakness. Many people with TK2d lose the ability to walk, eat and breathe independently. Management is primarily supportive care and there are no approved...