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Epigenetic Therapy for Friedreich Ataxia
OBJECTIVE: To investigate whether a histone deacetylase inhibitor (HDACi) would be effective in an in vitro model for the neurodegenerative disease Friedreich ataxia (FRDA) and to evaluate safety and surrogate markers of efficacy in a phase I clinical trial in patients. METHODS: We used a human FRDA...
Sparad:
I publikationen: | Ann Neurol |
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Huvudupphovsmän: | , , , , , , , , , , , , , , , , , , , , , |
Materialtyp: | Artigo |
Språk: | Inglês |
Publicerad: |
2014
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Ämnen: | |
Länkar: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4361037/ https://ncbi.nlm.nih.gov/pubmed/25159818 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ana.24260 |
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