UCB Publishes pyrimidine nucleo(s)(t)ide therapy results in TK2d in journal
UCB announced the publication of results from a multicenter retrospective chart review study of pyrimidine nucleo(s)(t)ide therapy in patients with thymidine kinase 2 deficiency (TK2d) in Neurology, which are in line with previous reports of the positive outcomes on survival and functional outcomes presented at a medical conference earlier this year. Note that TK2d is an ultra-rare genetic mitochondrial disease with no approved therapies, and has a prevalence of 1.64 per 1m people. Doxecitine and doxribtimine is currently under regulatory review in the US and EU, and a decision is expected by YE25. Given the ultra-rare nature of the disease, we model peak sales of € 150m by 2034. ACCUMULATE maintained.UCB is a global biopharmaceutical company focused on severe diseases in two therapeutic areas, namely central nervous system disorders and immunology. In the area of central nervous system disorders, Co. is focused on advanced Parkinson's disease, epilepsy, restless legs syndrome and epilepsy. In the area of immunology disorders, Co. is focused on rheumatoid arthritis, axial spondyloarthritis, psoriatic arthritis, systemic lupus erythematosus, juvenile idiopathic arthritis, post-menopausal osteoporosis, systemic lupus erythematosus and immunological diseases.
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