Friday, June 3, 2016

10 top patient groups oppose REGROW Act that would gut stem cell oversight

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The Niche, Knoepfler lab stem cell blog. June 1, 2016. The REGROW Act would drastically weaken FDA regulation of experimental stem cell th...
Thursday, June 2, 2016

US FDA Grants Orphan Drug Designation for Retrotope's RT001 in the Treatment of Friedreich's Ataxia

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LOS ALTOS, CA -- (Marketwired) -- 06/01/16 -- Retrotope announced today that the U.S. Food and Drug Administration (FDA) Office of Orphan Pr...
Wednesday, June 1, 2016

Epoetin Alpha Improves Upper-Limb Dexterity in Patients With Friedreich’s Ataxia: Presented at EAN

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FirstWord Pharma, By Chris Berrie. COPENHAGEN, Denmark -- May 31, 2016 -- Epoetin alpha does not modify frataxin levels or improve physica...
Tuesday, May 31, 2016

Paying for future success in gene therapy

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Stuart H. Orkin, Philip Reilly. Science 27 May 2016: Vol. 352, Issue 6289, pp. 1059-1061 DOI: 10.1126/science.aaf4770 OPEN The authors ...
Monday, May 30, 2016

Uncovering brain-heart information through advanced signal and image processing.

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Philos Trans A Math Phys Eng Sci. 2016 May 13; 374(2067): 20160020. doi: 10.1098/rsta.2016.0020 A recent article in Scientific American (...
Sunday, May 29, 2016

Inside HDACs with more selective HDAC inhibitors

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Joëlle Roche, Philippe Bertrand, European Journal of Medicinal Chemistry, Available online 25 May 2016, ISSN 0223-5234, doi: 10.1016/j.ejmec...
Saturday, May 28, 2016

Mitochondrial energy imbalance and lipid peroxidation cause cell death in Friedreich’s ataxia

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R Abeti, M H Parkinson, I P Hargreaves, P R Angelova, C Sandi, M A Pook, P Giunti and A Y Abramov. Cell Death and Disease (2016) 7, e2237; d...
Friday, May 27, 2016

Mutations in the substrate binding glycine-rich loop of the mitochondrial processing peptidase-α protein (PMPCA) cause a severe mitochondrial disease

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Mugdha Joshi, Irina Anselm, Jiahai Shi, Tejus A. Bale, Meghan Towne, Klaus Schmitz-Abe, Laura Crowley, Felix C. Giani, Shideh Kazerounian, K...
Thursday, May 26, 2016

JOT101, a product for treatment of Friedreich’s ataxia.

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Jupiter, Florida (PRWEB) May 25, 2016, by Christer Rosén, Jupiter Orphan Therapeutics. Jupiter Orphan Therapeutics, Inc. (JOT) today anno...
Wednesday, May 25, 2016

Purkinje cell injury, structural plasticity and fusion in patients with Friedreich’s ataxia

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Kevin C. Kemp, Amelia J. Cook, Juliana Redondo, Kathreena M. Kurian, Neil J. Scolding and Alastair Wilkins. Acta Neuropathologica Communicat...