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 ...
Friday, April 14, 2017

Australian children living with rare diseases: experiences of diagnosis and perceived consequences of diagnostic delays

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Yvonne Zurynski, Marie Deverell, Troy Dalkeith, Sandra Johnson, John Christodoulou, Helen Leonard, Elizabeth J Elliott and APSU Rare Disease...
Thursday, April 13, 2017

No changes in heme synthesis in human Friedreich´s ataxia erythroid progenitor cells

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Hannes Steinkellner, Himanshu Narayan Singh, Martina U. Muckenthaler, Hans Goldenberg, Rajeswari R. Moganty, Barbara Scheiber-Mojdehkar, Bri...
Wednesday, April 12, 2017

Drugmakers accused of exploiting orphan drug incentives, fueling price problem

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Brady Huggett, Nature Biotechnology 35, 301 (2017) doi:10.1038/nbt0417-301 Published online 11 April 2017 n January Kaiser Health News pub...
Tuesday, April 11, 2017

Biogen Accused Of Stifling Neurological Drug Competition

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Law360, Los Angeles (April 4, 2017, 7:05 PM EDT) -- Biogen Inc. is facing an antitrust suit in California federal court filed Tuesday by Ixc...
Saturday, April 8, 2017

SNPs in microRNA target sites and their potential role in human disease

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Adrianna Moszyńska, Magdalena Gebert, James F. Collawn, Rafał Bartoszewski; Open Biology, The Royal Society Publishing, Published 5 April 20...