Wednesday, October 18, 2017

BioMarin Selects BMN 290 for Friedreich's Ataxia

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SAN RAFAEL, Calif., Oct. 18, 2017 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) updated the investment community on the Compan...

New data for Friedreich's ataxia with a novel capsid demonstrate reversal of disease phenotype in a preclinical disease model.

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Press Release: CAMBRIDGE, Mass., Oct. 17, 2017 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (NASDAQ:VYGR), a clinical-stage gene therapy c...
Tuesday, October 17, 2017

Recombinant adeno-associated viral vector serotype 5 carrying the gene for the human frataxin protein for the treatment of Friedreich’s ataxia

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EU/3/17/1906: Public summary of opinion on orphan designation (First published: 17/10/2017): On 23 August 2017, orphan designation (EU/3/17/...

Therapies for mitochondrial diseases and current clinical trials

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Ayman W. El-Hattab, Ana Maria Zarante, Mohammed Almannai, Fernando Scaglia, In Molecular Genetics and Metabolism, 2017, , ISSN 1096-7192, do...
Monday, October 16, 2017

In Vivo Assessment of Mitochondrial Dysfunction in Clinical Populations Using Near-Infrared Spectroscopy

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T. Bradley Willingham and Kevin K. McCully, Front. Physiol., 14 September 2017 doi:10.3389/fphys.2017.00689 The ability to sustain submaxi...
Sunday, October 15, 2017

Estudos moleculares em ataxia de Friedreich

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Peluzzo, Thiago Mazzo, Advisor: França Junior, Marcondes Cavalcante; TESE DIGITAL 2017 We therefore designed this study to determine the f...
Friday, October 13, 2017

CRISPR Therapeutics Awarded Grant from Friedreich’s Ataxia Research Alliance to Collaborate with University of Alabama at Birmingham on Gene-edited Treatments for Friedrich’s Ataxia

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ZUG, Switzerland and CAMBRIDGE, Mass., Oct. 13, 2017 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ:CRSP), a genome editing company focused...

FDA awards six grants for natural history studies in rare diseases

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12-10-2017. SILVER SPRING, Md: The U.S. Food and Drug Administration today announced it has awarded six new research grants for natural hist...
Wednesday, October 11, 2017

Mathematical modeling of Friedreich’s ataxia – a genetic neuro-muscular degenerative condition

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SWASTI WAGH and D.K. WAGH; Journal of Ultra Scientist of Physical Sciences, Volume 29, Issue 2, Page Number 66-70, 2017 doi:10.22147/jusps-A...
Tuesday, October 10, 2017

The New Zealand Neuromuscular Disease Patient Registry; Five Years and a Thousand Patients.

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Rodrigues, Miriam J., O’Grady, Gina, Hammond-Tooke, Graemed, Kidd, Alexaf, Love, Donald O.g, Baker, Ronelle K.b, Roxburgh, Richard H.a; Jour...