Application de la méthodologie bayésienne a un essai clinique de petit effectif. Exemple de l’essai ACTFRIE : étude de l’effet de la pioglitazone dans l’ataxie de Friedreich

Application de la méthodologie bayésienne a un essai clinique de petit effectif. Exemple de l’essai ACTFRIE : étude de l’effet de la pioglitazone dans l’ataxie de Friedreich. B. Andriss, I. Husson, S. Guilmin Crepon, E. Jacqz-Aigrain, S. Zohar, C. Alberti; Revue d'Épidémiologie et de Santé Publique, Volume 63, Supplement 2, May 2015, Page S63, ISSN 0398-7620, http://dx.doi.org/10.1016/j.respe.2015.03.058.
(http://www.sciencedirect.com/science/article/pii/S0398762015002047)

Study of beta cells and neurons indicate incretin analogs as potential therapeutics for Friedreich’s ataxia

Study of beta cells and neurons indicate incretin analogs as potential therapeutics for Friedreich’s ataxia. Massimo Pandolfo, Mariana Igoillo-Esteve, Amélie Hu, Ewa Gurgul-Convey, Laila Romagueira Bichara Dos Santos, Jonas Jean-Christophe, Decio Eizirik, and Miriam Cnop; Neurology April 6, 2015 84:14 Supplement S32.005; 1526-632X

Magnetic Resonance is more sensitive than Echocardiography to detect Cardiomyopathy in Friedreich’s Ataxia.

Magnetic Resonance is more sensitive than Echocardiography to detect Cardiomyopathy in Friedreich’s Ataxia. Alberto Martinez, Ingrid Faber, Thiago Rezende, Cynthia Bonilha Da Silva, Thiago Venâncio, Anelyssa D'Abreu, Iscia Lopes-Cendes, Otávio Coelho-Filho, and Marcondes Franca, Jr.; Neurology April 6, 2015 84:14 Supplement P2.121; 1526-632X

Diagnosis of Friedreich’s Ataxia in Late-Onset Progressive Cerebellar Ataxia

Diagnosis of Friedreich’s Ataxia in Late-Onset Progressive Cerebellar Ataxia. Roisin Lonergan, Damien Ferguson * Joint first author with RL, Susan Byrne, David Bradley, Yvonne Langan, and Janice Redmond; Neurology April 6, 2015 84:14 Supplement P2.119; 1526-632X

Gene Expression Profile in Peripheral Blood Cells of Friedreich Ataxia Patients

Gene Expression Profile in Peripheral Blood Cells of Friedreich Ataxia Patients. Agessandro Abrahao, Jose Luiz Pedroso, Patricia de Carvalho Aguiar, and Orlando Barsottini; Neurology April 6, 2015 84:14 Supplement P2.122; 1526-632X

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)