⌘+K

Pulse Explore Communities Researchers Journals

Home Communities Explore

July 18, 2024EClinicalMedicineOpen Access

Safety and efficacy of deoxycytidine/deoxythymidine combination therapy in POLG-related disorders: 6-month interim results of an open-label, single arm, phase 2 trial

Heather Pekeles Saoussen Berrahmoune Christelle Dassi Anthony C T Cheung Tommy Gagnon Paula J. Waters Ralf Eberhard Daniela Buhaş Kenneth A. Myers

View Full Paper

Ask AI

Bookmark

Share

Abstract

DNA polymerase gamma (POLG)-related disorders are a group of rare neurodegenerative mitochondrial diseases caused by pathogenic variants in

Discussion

No takes yet. Share an insight, caveat, or question.

Cite This Study

Pekeles et al. (2024) studied this question.

synapsesocial.com/papers/68e5fda8b6db643587591128 https://doi.org/10.1016/j.eclinm.2024.102740

View Full Paper

Ask AI

Bookmark

Share

Keep reading

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

1Long-term safety and efficacy of deoxycytidine/deoxythymidine in treatment of POLG-related disordersEarly-Phase Clinical Trial · 2026 · 2 citations
2Liver transplantation for valproic acid-induced acute liver failure in POLG mutation-related mitochondrial disease with cardiac and neurological involvementOriginal Research · 2026
3Juvenile/adult-onset <i>POLG</i> -related disease unmasked by valproate-associated fulminant hepatic failure2025 · 1 citations
4Impaired NADH-linked mitochondrial respiration disrupts ventral midbrain neuronal programs in POLG diseaseOriginal Research · 2026
5A Rare Molecular Diagnosis in a Patient With Hepatocerebral Syndrome Contributes to the Expansion of the Phenotypic Spectrum of <scp> <i>POLG2</i> </scp> ‐Related Mitochondrial DisorderOriginal Research · 2025

Efficacy and safety of pyrimidine nucleos(t)ide therapy in thymidine kinase 2 deficiency2026
Hallmark Molecular and Pathological Features of POLG Disease are Recapitulated in Cerebral Organoids2024
Association of Mitochondrial DNA Polymerase γ Gene POLG1 Polymorphisms with Parkinsonism in Chinese Populations2012
Early best available antibiotic therapy for multidrug-resistant gram-negative bloodstream infections: a target trial emulationEClinicalMedicine · 2026
Single-dose/day versus extended antibiotic prophylaxis for elective urological surgery: a prospective randomized controlled trialEClinicalMedicine · 2026

More from EClinicalMedicine

More from McGill University

More from McGill University Health Centre

More from Université de Sherbrooke

More from Montreal Children's Hospital

More from Centre Hospitalier Universitaire de Sherbrooke