Correlation of GAITRite Walkway System and Biodex Balance System Measures to the FARS Score in Friedreich's Ataxia Patients: A Validation Study

Correlation of GAITRite Walkway System and Biodex Balance System Measures to the FARS Score in Friedreich's Ataxia Patients: A Validation Study. Theresa Zesiewicz, Seok Kim, Clifton Gooch, Jennifer Farmer, Yangxin Huang, Jason Salemi, Israt Jahan, Kelly Sullivan, Lynn Wecker, and Jeannie Stephenson; Neurology April 6, 2015 84:14 Supplement P2.123; 1526-632X

Longitudinal Study of Gait Dysfunction in Friedreich's Ataxia Using the GAITRite Walkway System

Longitudinal Study of Gait Dysfunction in Friedreich's Ataxia Using the GAITRite Walkway System. Theresa Zesiewicz, Clifton Gooch, Seok Kim, Jennifer Farmer, Israt Jahan, Yangxin Huang, Jason Salemi, Kelly Sullivan, and Jeannie Stephenson; Neurology April 6, 2015 vol. 84 no. 14 Supplement P2.120

Progressive Pyramidal And Callosal Damage In Friedreich’s Ataxia

Progressive Pyramidal And Callosal Damage In Friedreich’s Ataxia (P2.124). Thiago Rezende, Cynthia Silva, Alberto Martinez, Anelyssa D'Abreu, Iscia Lopes-Cendes, Fernando Cendes and Marcondes Franca, Jr.. Neurology April 6, 2015 vol. 84 no. 14 Supplement P2.124

Open-label Pilot Study of Methylprednisolone for the Treatment of Patients With Friedreich Ataxia (FRDA)

Open-label Pilot Study of Methylprednisolone for the Treatment of Patients With Friedreich Ataxia (FRDA). Sponsor: Children's Hospital of Philadelphia, Collaborator: Friedreich's Ataxia Research Alliance. First received: April 16, 2015

This study will explore whether methylprednisolone treatment is safe, well-tolerated, and beneficial in patients that are diagnosed with Friedreich Ataxia (FRDA). The study will also explore if methylprednisolone has any effects on biomarkers associated with FRDA. All subjects in the study will receive the same steroid treatment.
This study will take place at the Children's Hospital of Philadelphia as an outpatient trial in 5 children who are at least 5 years and less than 10 years of age, and in 5 adults ages 45 years and older, with genetically confirmed FRDA.

German regulator puts UniQure gene therapy appraisal on hold

German regulator puts UniQure gene therapy appraisal on hold. REUTERS. FRANKFURT, April 17

(Reuters) - A German regulator for new drugs has suspended the assessment of the Western world's first gene therapy, UniQure's Glybera, after an adviser to the European drugs watchdog voiced concern over the treatment. (Read more)

Robotic and clinical evaluation of upper limb motor performance in patients with Friedreich’s Ataxia: an observational study

Robotic and clinical evaluation of upper limb motor performance in patients with Friedreich’s Ataxia: an observational study. Marco Germanotta, Gessica Vasco, Maurizio Petrarca, Stefano Rossi, Sacha Carniel, Enrico Bertini, Paolo Cappa and Enrico Castelli; Journal of NeuroEngineering and Rehabilitation 2015, 12:41 doi:10.1186/s12984-015-0032-6

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First Presentation of Diabetes as Diabetic Ketoacidosis in a Case of Friedreich’s Ataxia

First Presentation of Diabetes as Diabetic Ketoacidosis in a Case of Friedreich’s Ataxia. Partha Pratim Chakraborty, Sayantan Ray, Rana Bhattacharjee, Sujoy Ghosh, Pradip Mukhopadhyay, Satinath Mukhopadhyay and Subhankar Chowdhury; Clinical Diabetes April 2015 vol. 33 no. 2 84-86, doi: 10.2337/diaclin.33.2.84

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Measuring Disease Progression in Giant Axonal Neuropathy: Implications for Clinical Trial Design

Measuring Disease Progression in Giant Axonal Neuropathy: Implications for Clinical Trial Design ; Lisa A. Roth, MD, MS,
Jonathan D. Marra, MA, Nicole H. LaMarca, DNP, MSN, CPNCJ; Child Neurol May 2015 30: 741-748 doi: 10.1177/0883073814542946

As part of a natural history study of giant axonal neuropathy, we hypothesized that the Friedreich Ataxia Rating Scale and the Gross Motor Function Measure would show a significant change over 6 months ....

Positive Preclinical Proof-of-Concept Results of BB-FA in Mice Model of Friedreich’s Ataxia

Positive Preclinical Proof-of-Concept Results of BB-FA in Mice Model of Friedreich’s Ataxia. BioBlast Announced Preclinical Data and Pipeline Updates. April 14, 2015

BioBlast (NasdaqGM: ORPN) announced positive in vitro and in vivo results from a therapy for Friedreich’s ataxia based on the mitochondria-targeting platform, providing support for further clinical development.

Emerging functional cross-talk between the Keap1-Nrf2 system and mitochondria

Emerging functional cross-talk between the Keap1-Nrf2 system and mitochondria. Ken Itoh,, Peng Ye, Tomoh Matsumiya, Kunikazu Tanji, and Taku Ozaki; J Clin Biochem Nutr. 2015 March; 56(2): 91–97. doi: 10.3164/jcbn.14-134

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