Long-term safety and efficacy of deoxycytidine/deoxythymidine in treatment of POLG-related disorders

We evaluated the safety and efficacy of enteral deoxycytidine/deoxythymidine combination therapy in treatment of POLG-related disorders, genetic mitochondrial diseases characterized by progressive neurological degeneration. A single-centre open-label phase II trial was conducted. Inclusion criteria...

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Hlavní autoři:	Anthony C.T. Cheung, Saoussen Berrahmoune, Christelle Dassi, Heather Pekeles, Tommy Gagnon, Paula J. Waters, Ralf Eberhard, Daniela Buhas, Kenneth A. Myers
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	Elsevier 2026-07-01
Edice:	Neurotherapeutics
Témata:	DNA polymerase gamma POLG Deoxynucleoside Mitochondrial disorder Mitochondrial DNA depletion disorder
On-line přístup:	http://www.sciencedirect.com/science/article/pii/S1878747926001157
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