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New data on investigational therapy for thymidine kinase 2 deficiency presented at Muscular Dystrophy Association (MDA) 2025 Conference [Video]

  • Clinical data highlight survival benefits and improvement in functional motor outcomes associated with treatment with doxecitine (dC) and doxribtimine (dT) – an investigational pyrimidine nucleoside therapy – in people living with thymidine kinase 2 deficiency (TK2d).1,2,3
  • Additional patient experience data emphasize the profound physical challenges and severe psychological strain that come with living with TK2d, and the heavy emotional and physical burden experienced by caregivers.4,5
  • Across all study populations, pyrimidine nucleoside and/or nucleotide therapy was generally well tolerated.1,2,3
  • TK2d is an ultra-rare, life-threatening, genetic mitochondrial disease with no currently approved treatment options.6,7,8,9,10,11

BRUSSELS, March 19, 2025 /PRNewswire/ — UCB, a global biopharmaceutical company, today announced positive data from studies involving its investigational pyrimidine nucleoside therapy, doxecitine (dC) and doxribtimine (dT), in people living with thymidine kinase 2 deficiency (TK2d), at this year’s MDA Clinical and Scientific Conference, Dallas, Texas, March 16-19, 2025.

The data show that …

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