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UnknownEfficacy and Safety of Leriglitazone in Patients With Friedreich Ataxia: A Phase 2 Double-Blind, Randomized Controlled Trial (FRAMES).
Not available
S-EPMC9747094 | biostudies-literature
UnknownThe effects of somatostatin analogues on liver volume and quality of life in polycystic liver disease: a meta-analysis of randomized controlled trials.
Not available
S-EPMC8648823 | biostudies-literature
TranscriptomicsEpigenetic therapy for Friedreich ataxia.
We set out 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. In the neuronal cell model, H...
ORGANISM(S): Homo sapiens 
2015-01-29 | E-GEOD-65399 | biostudies-arrayexpress
TranscriptomicsTranscription profiling of ataxin-null (Atxn-null) vs. wild type mice to investigate spinocerebellar ataxia type 1 (SCA1)
Ataxin 1 (Atxn1) is a protein of unknown function associated with cerebellar neurodegeneration in spinocerebellar ataxia type 1 (SCA1). SCA1 is caused by an expanded polyglutamine within Atxn1 by gain-of-function mechanisms. Lack of Atxn1 in mice triggers motor deficits in the absence of neurodegene...
ORGANISM(S): Mus musculus 
2006-12-01 | E-MEXP-886 | biostudies-arrayexpress
TranscriptomicsFunctional genomic analysis of frataxin deficiency, Illumina data
Functional genomic analysis of frataxin deficiency reveals tissue-specific alterations and identifies the PPARγ pathway as a therapeutic target in Friedreich's ataxia Friedreich's ataxia (FRDA), the most common inherited ataxia, is characterized by focal neurodegeneration, diabetes mellitus, and l...
ORGANISM(S): Mus musculus 
2010-04-09 | E-GEOD-15848 | biostudies-arrayexpress
TranscriptomicsFunctional genomic analysis of frataxin deficiency, Agilent data
Functional genomic analysis of frataxin deficiency reveals tissue-specific alterations and identifies the PPARγ pathway as a therapeutic target in Friedreich's ataxia Friedreich's ataxia (FRDA), the most common inherited ataxia, is characterized by focal neurodegeneration, diabetes mellitus, and li...
ORGANISM(S): Mus musculus 
2009-06-02 | E-GEOD-15843 | biostudies-arrayexpress
UnknownReal-time PCR and linkage studies to identify carriers presenting HPRT deleted gene.
Not available
S-EPMC1770009 | biostudies-literature
UnknownPharmacological treatments for Friedreich ataxia.
Not available
S-EPMC6457808 | biostudies-literature
UnknownPrimary proprioceptive neurons from human induced pluripotent stem cells: a cell model for afferent ataxias.
Not available
S-EPMC7210273 | biostudies-literature
UnknownCerebrospinal Fluid Proteomics in Friedreich Ataxia Reveals Markers of Neurodegeneration and Neuroinflammation.
Not available
S-EPMC9326443 | biostudies-literature
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