Leading the worldwide fight to treat and cure Tay-Sachs, Canavan, GM1 and Sandhoff diseases
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1940 downloads
Poster Futerman.pdf
1870 downloads
Layman progress report_Jiang(Aug 2019 - Jan 2020).pdf
1863 downloads
NTSAD2021_summary_d'Azzo.pdf
2080 downloads
Sanofi Genzyme GM2 Patient Journey Poster.pdf
1644 downloads
2019 September NTSAD d'Azzo_Lay summary_ 2019.pdf
Role of Plasma membrane-ER Contact Sites in GM1-mediated Neuronal Cell Death
Alessandra d’AzzoDepartment of GeneticsSt. Jude Children’s Research Hospital,Memphis TN
1762 downloads
2019 September Gray-Edwards NTSAD Lay Summary 0919.pdf
Minimally invasive delivery of AAV gene therapy in the Tay-Sachs sheep
The goal is to determine the efficacy of AAV gene therapy using established biomarkers after CSF delivery.
Heather Gray-Edwards, PhD, DVMDepartment of RadiologyUMass Medical School
1689 downloads
2019 September Lay progress report_Jiang 12 months.pdf
Oligosaccharide Biomarkers for Disease Progression and AAV Therapeutic Efficacy inGM1Gangliosidosis
Modified on 13 September 2019 1779 downloads
Poster Presentation_AAN_5-19.pdf
At the May 2019 American Academy of Neurology Scientific Sessions, Dr. Robert Molinari presented Retrotope’s poster presentation entitled: “RT001 to Treat Neurodegeneration--Case Study: Improvement in a Single Patient with Late Onset Tay-Sachs Disease (LOTS).RT001 is a di-deuterated isotope of linoleic acid (D-LA) that is readily incorporated into membranes and prevents oxidative damage.
3835 downloads
Natural History of LOTS - LDN poster - Eichler - Feb, 2012.pdf
3753 downloads
Poster_Session_DP2_Gangliosides.pdf
Modified on 06 June 2014 4339 downloads
Poster_Session_PSM06_Metabolism.pdf
Plenary Talk P01: Experience-dependent modification of neural circuits – cellular and molecular mechanism
Modified on 06 June 2014 3452 downloads
ABSTRACT_NextGen-Sequencing-HEXA.pdf
NextGen Sequencing of HEXA: A more sensitive assay
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