Monday, May 1, 2017

The new top 10 most expensive drugs on the planet

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Endpoints News. by John Carroll April 28, 2017 07:38 AM ¨5. Actimmune $572,292¨ Company: Horizon Category: Rare disease – orphan drug...
Sunday, April 30, 2017

Mesenchymal Stem Cell-Derived Factors Restore Function to Human Frataxin-Deficient Cells

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Kevin Kemp Rimi Dey, Amelia Cook, Neil Scolding, Alastair Wilkins; Cerebellum (2017). doi:10.1007/s12311-017-0860-y The demonstration that...
Saturday, April 29, 2017

Frataxin Deficiency Impairs Mitochondrial Biogenesis in Cells, Mice and Humans

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Mittal J. Jasoliya, Marissa Z. McMackin, Chelsea K. Henderson, Susan L. Perlman, Gino A. Cortopassi; Hum Mol Genet 2017 ddx141. doi: 10.1093...
Friday, April 28, 2017

Dimethyl Fumarate Mediates Nrf2-dependent Mitochondrial Biogenesis in Mice and Humans

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Genki Hayashi, Mittal Jasoliya, Francesco Saccà, Chiara Pane, Alessandro Filla, Angela Marsili, Giorgia Puorro, Roberta Lanzillo, Vincenzo B...
Wednesday, April 26, 2017

Retrotope To Present First Human Data On Safety And Early Efficacy Of RT001 With Neurodegeneration At American Academy of Neurology Meeting

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LOS ALTOS, CA, April 21, 2017 – Dr. Theresa Zesiewicz, University of South Florida Movement Disorders Clinic, will give a podium presentatio...
Tuesday, April 25, 2017

Determinants of orphan drugs prices in France: a regression analysis

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Daria Korchagina, Aurelie Millier, Anne-Lise Vataire, Samuel Aballea, Bruno Falissard and Mondher Toumi; Orphanet Journal of Rare Diseases 2...
Monday, April 24, 2017

CA Cycle in the Dentate in Friedreich's Ataxia

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ClinicalTrials.gov Identifier: NCT03122925 First received: April 18, 2017 OBJECTIVE: To measure the tricarboxylic acid (TCA) cycle rate in...
Friday, April 21, 2017

Orphan Drugs: Getting Arms around Rare Diseases

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Duygu Koyuncu Irmak; J Comm Pub Health Nurs 2017, 3:2 DOI: 10.4172/2471-9846.1000167 Finding ways to bring new therapies for rare diseases...
Thursday, April 20, 2017

Rehabilitative Trial With Cerebello-Spinal tDCS in Neurodegenerative Ataxia (CStDCSAtaxia)

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ClinicalTrials.gov Identifier: NCT03120013 First received: April 14, 2017 Neurodegenerative cerebellar ataxias represent a group of disabl...
Saturday, April 15, 2017

Friedreich Ataxia: current status and future prospects

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Bürk K; Cerebellum Ataxias. 2017 Apr 7;4:4. doi: 10.1186/s40673-017-0062-x. eCollection 2017. This review gives an overview over clinical ...