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Leading the Fight to treat and cure
Tay-Sachs, Canavan, Sandhoff, GM1 and related diseases

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pdf Cerebral organoids derived from Sandhoff disease induced pluripotent stem cells exhibit impaired neurodifferentiation ( pdf, 3.94 MB ) (197 downloads) Popular
pdf Intravenous administration of scAAV9-Hexb normalizes lifespan and prevents pathology in Sandhoff disease mice ( pdf, 3.37 MB ) (284 downloads) Popular
pdf Haematopoietic Stem Cell Transplantation Arrests the Progression of Neurodegenerative Disease in Late-Onset Tay-Sachs Disease ( pdf, 145 KB ) (366 downloads) Popular
pdf Intracerebroventricular delivery of hematopoietic progenitors results in rapid and robust engraftment of microglia-like cells ( pdf, 569 KB ) (291 downloads) Popular
pdf Families and Researchers Collaborate in Advancing Therapy: The Role of NTSAD, Together with Parents, in Driving Toward Treatments ( pdf ) (661 downloads) Popular
pdf Effect of cyclic, low dose pyrimethamine treatment in patients with Late Onset Tay Sachs: an open label, extended pilot study ( pdf, 628 KB ) (1614 downloads) Popular
pdf European-Journal-of-Medical-Genetics_Miglustat-Ketogenic-Sandhoff ( pdf, 872 KB ) (1924 downloads) Popular
pdf Science Trans-Medicine Long-term Follow-up for GT for Canavan Leone ( pdf, 3.42 MB ) (2259 downloads) Popular
pdf PNAS Cachon Gonzalez et al TS Effective Gene Therapy 12 2006 ( pdf, 595 KB ) (1246 downloads) Popular
pdf PLoS Mice Lysosomal HexA ( pdf, 3.11 MB ) (1206 downloads) Popular
pdf PLoS Novel Patient Cell Based HTS Assay Small Molecules Maegawa 2011 ( pdf, 1.48 MB ) (1011 downloads) Popular
pdf Nature Medicine 2007 Stem Cells Multiple Mechanisms Lee Seyfried Platt Snyder ( pdf, 581 KB ) (1322 downloads) Popular
pdf Neurology 2006 LOTS Adverse Effects Medications Shapiro ( pdf, 96 KB ) (1857 downloads) Popular
pdf Natural History of Infantile GM2 Gangliosidosis, Pediatrics, Bley et al 2011 95 95 ( pdf, 551 KB ) (979 downloads) Popular
pdf Nature America GeneTherapy Biotechnology article 07 2012 ( pdf, 234 KB ) (852 downloads) Popular
pdf Molecular Brain Research Kyrkanides et al 2005 ( pdf, 846 KB ) (1529 downloads) Popular
pdf Molecular Genetics and Metabolism Jan 2011 Pyrimethamine Clarke ( pdf, 340 KB ) (1602 downloads) Popular
pdf Journal of Neuroimmunology Pathway Treatment Neuro Kyrkanides 2009 ( pdf, 1.08 MB ) (1292 downloads) Popular
pdf Human Molecular Genetics Combo therapies for LSD Hawkins Sands ( pdf, 129 KB ) (1078 downloads) Popular
pdf Journal of Neurochemistry GM1 Platt Seyfried Kasperzyk ( pdf, 140 KB ) (921 downloads) Popular
pdf ASGCT Cachon Gonzalez et al 2012 GM2 Enzyme Flow ( pdf, 790 KB ) (1325 downloads) Popular
pdf Lysosomal Storage Disease Research Consortium Funded projects ( pdf, 69 KB ) (1391 downloads) Popular

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