Wednesday, June 8, 2016

Histone deacetylase inhibitors modulating non-epigenetic players: The novel molecular targets for therapeutic intervention

›
Shabir Ahmad Ganai. Current Drug Targets, Volume 17 DOI: 10.2174/1389450117666160527143257 Emerging evidences suggest that acetylation of ...
Tuesday, June 7, 2016

Quantifying benefit-risk preferences for new medicines in rare disease patients and caregivers

›
T. Morel, S. Aymé, D. Cassiman, S. Simoens, M. Morgan and M. Vandebroek. Orphanet Journal of Rare Diseases 2016 DOI: 10.1186/s13023-016-044...
Monday, June 6, 2016

The hereditary ataxias: Where are we now? Four decades of local research

›
D C Smith, L J Greenberg, A Bryer. South African Medical Journal 2016;106(6):S38. DOI:10.7196/SAMJ.2016.v106i6.10989 The hereditary ataxia...
Sunday, June 5, 2016

The significance of intercalated discs in the pathogenesis of Friedreich cardiomyopathy

›
Arnulf H. Koeppen, Alyssa B. Becker, Paul J. Feustel, Benjamin B. Gelman, Joseph E. Mazurkiewicz. Journal of the Neurological Sciences, Avai...
Saturday, June 4, 2016

Aspects éthico-sociétaux liés à l’utilisation de la technique d’édition du génome CRISPR-Cas9 (FR)

›
INSERM (Institut National de la santé et de la recherche médicale). Comité éthique Inserm février 2016. Saisine concernant les questions l...
Friday, June 3, 2016

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

›
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

›
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

›
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

›
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.

›
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 (...