Emergency Use Protocol for EPI-743 in Acutely Ill Patients With Inherited Mitochondrial Respiratory Chain Disease Within 90 Days of End-of-Life Care

This study is currently recruiting participants.

First Received on June 7, 2011. Last Updated on June 9, 2011
Sponsor: Edison Pharmaceuticals Inc
Information provided by: Edison Pharmaceuticals Inc
ClinicalTrials.gov Identifier: NCT01370447


Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Transcranial Sonography Reveals Cerebellar, Nigral, and Forebrain Abnormalities in Friedreich’s Ataxia

Neurodegenerative Dis, (DOI: 10.1159/000327751)

Matthis Synofzik, Jana Godau, Tobias Lindig, Ludger Schöls, Daniela Berg
Department of Neurodegenerative Diseases, Hertie Institute for Clinical Brain Research, and German Research Center for Neurodegenerative Diseases, University of Tübingen, Tübingen, Germany

Keywords: Ataxia, Transcranial sonography, Friedreich’s ataxia, Imaging, Substantia nigra, Dentate nucleus, Iron, Cerebellum

Mortality in Friedreich Ataxia

Journal of the Neurological Sciences,Article in Press,
doi:10.1016/j.jns.2011.05.023

(J Neurol Sci. 2011 Aug 15;307(1-2):46-9. Epub 2011 Jun 8.)

Amy Y. Tsoua, Erin K. Paulsena, b, c, d, Sarah J. Lagedrosta, b, c, d, Susan L. Perlmane, Katherine D. Mathewsf, George R. Wilmotg, Bernard Ravinah, Arnulf H. Koeppeni, j and David R. Lyncha, b, c, d,

a Department of Neurology, University of Pennsylvania Medical School, United States
b Department of Pediatrics, University of Pennsylvania Medical School, United States
c Children's Hospital of Philadelphia, United States
d University of Pennsylvania Medical School, United States
e University of California Los Angeles, Los Angeles, CA, United States
f University of Iowa, Iowa City, IA, United States
g Emory University, Atlanta, GA, United States
h University of Rochester, Rochester, NY, United States
i VA Hospital, Albany, NY, United States

Keywords: Cerebellum; Sensory; Cardiomyopathy; Echocardiogram; Arrhythmia; Neurodegenerative disease