Use of MAPK pathway inhibitors for the treatment of Friedreich Ataxia . University of Pennsylvania, posted on 05/19/2015
Brief Description: A novel treatment for rare disease Friedreich ataxia using p38 or MK2 kinase inhibitors
Wednesday, May 20, 2015
A First in Human Study of RT001 in Patients With Friedreich's Ataxia
A First in Human Study of RT001 in Patients With Friedreich's Ataxia.ClinicalTrials.gov Identifier: NCT02445794, Sponsor: Retrotope, Inc.
Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Estimated Enrollment: 18
Study Start Date: July 2015
Estimated Study Completion Date: February 2016
Estimated Primary Completion Date: January 2016 (Final data collection date for primary outcome measure)
Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Estimated Enrollment: 18
Study Start Date: July 2015
Estimated Study Completion Date: February 2016
Estimated Primary Completion Date: January 2016 (Final data collection date for primary outcome measure)
The Cardiomyopathy in Friedreich’s Ataxia – New Biomarker for Staging Cardiac Involvement
The Cardiomyopathy in Friedreich’s Ataxia – New Biomarker for Staging Cardiac Involvement. Frank Weidemann, Dan Liu, Kai Hu, Cristiane Florescu, Markus Niemann, Sebastian Herrmann, Bastian Kramer, Stephan Klebe, Kathrin Doppler, Nurcan Üçeyler, Christian Oliver Ritter, Georg Ertl, Stefan Störk, International Journal of Cardiology, Available online 15 May 2015, ISSN 0167-5273, http://dx.doi.org/10.1016/j.ijcard.2015.05.074.
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FULL TEXT PDF
A biomarker for determining mitochondrial damage in friedreich's ataxia
A biomarker for determining mitochondrial damage in friedreich's ataxia. Patent WO 2013130964, Pub. No.: WO/2013/130964, International Application No.: PCT/US2013/028609, Publication Date: 06.09.2013, International Filing Date: 01.03.2013
Applicants:INDIANA UNIVERSITY RESEARCH & TECHNOLOGY CORPORATION
Inventors: PAYNE, Ronald Mark; (US), WAGNER, Gregory R.; (US), BABBEY, Clifford M.; (US), PRIDE, P. Melanie; (US)
The compositions and methods include determining the acetylation status of mitochondrial proteins
Applicants:INDIANA UNIVERSITY RESEARCH & TECHNOLOGY CORPORATION
Inventors: PAYNE, Ronald Mark; (US), WAGNER, Gregory R.; (US), BABBEY, Clifford M.; (US), PRIDE, P. Melanie; (US)
The compositions and methods include determining the acetylation status of mitochondrial proteins
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