Patients in the #EU with rare disease #thymidinekinase2deficiency (TK2d) have their first approved therapy, #UCB's #Kygevi, after the #EuropeanCommission cleared the drug "under exceptional circumstances."

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Case Report: Novel Homozygous POLG Mutation A case report describes a novel homozygous POLG mutation, an important differential diagnosis for thymidine kinase 2 deficiency (TK2d).

A case involving a patient with a novel homozygous #POLG mutation, an important differential diagnosis for thymidine kinase 2 deficiency (#TK2d). Published in Acta Neurologica Belgica.

Read more: https://bit.ly/47V8qkp

#RareDisease #ThymidineKinase2Deficiency #MedSky #Neurology

MHRA grants Promising Innovative Medicine designation to UCB’s DoxTM for TK2d - PharmaTimes UCB’s developmental treatment may be eligible for patient access

#rarediseases #MHRA #UK #PIMdesignation #UCB #DoxTM #TK2d #ThymidineKinase2Deficiency #TK2Deficiency #patientaccess #TK2geneticmutation #mitochondrialDNAdepletion #EarlyAccesstoMedicines #EAMS #MitochondrialMedicine #Mitochondrialdiseases
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