Leading the worldwide fight to treat and cure
Tay-Sachs, Canavan, GM1 and Sandhoff diseases

Research

Research abstracts, publications, NTSAD initiatives, and Tay Sachs Gene Therapy Consortium (TSGT)

Categories

Folder NTSAD Annual Family Conference Research Presentations

This folder contains slide presentations, handouts and other materials relating to the Research Update session at the NTSAD Annual Family Conferences. Please do not alter, publish or copy without permission from NTSAD. 

Folder Subcommittee on Experimental Therapies

The goal of the subcommittee is to evaluate clinical trials or other proposed therapies for Tay-Sachs, GM1, Sandhoff, Canavan and related diseases to educate families regarding trials and how to assess a proposed new or untested therapy. Papers written on different topics of therapies and/or clinical trials will be saved in this folder.

Folder Tay-Sachs Gene Therapy

Semi-annual progress reports published by TSGC on gene therapy research.

Documents

pdf AC 121 21 NTSAD Website PDFS Phil R5 Popular

542 downloads

Download (pdf, 1.23 MB)

AC-121-21 NTSAD Website PDFS Phil R5.pdf

pdf AC 121 21 NTSAD Website PDFS Vayle R5 Popular

334 downloads

Download (pdf, 1.00 MB)

AC-121-21 NTSAD Website PDFS Vayle R5.pdf

pdf AC 121 21 NTSAD Website PDFS Elliott R6 Popular

226 downloads

Download (pdf, 971 KB)

AC-121-21 NTSAD Website PDFS Elliott R6.pdf

pdf AC 121 21 NTSAD Website PDFS Embree R5 Popular

227 downloads

Download (pdf, 1.45 MB)

AC-121-21 NTSAD Website PDFS Embree R5.pdf

pdf AC 121 21 NTSAD Website PDFS Joey R5 Popular

211 downloads

Download (pdf, 1.25 MB)

AC-121-21 NTSAD Website PDFS Joey R5.pdf

pdf Intrabio Reports Further Detail On Positive Data From IB1001 Multinational Clinical Trial For The Treatment of GM2 Gangliosidosis Popular

281 downloads

Download (pdf, 175 KB)

Intrabio Reports Further Detail On Positive Data From IB1001 Multinational Clinical Trial For The Treatment of GM2 Gangliosidosis.pdf

pdf AXO AAV GM1 Trial Brochure Jun2021 Popular

393 downloads

Download (pdf, 1.10 MB)

AXO-AAV-GM1-Trial-Brochure-Jun2021.pdf

pdf GM2 Queens Recrutiment REB Submission FINAL Brochure Popular

432 downloads

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GM2 Queens Recrutiment_REB Submission_FINAL_Brochure.pdf

pdf AXO AAV GM2 Trial Brochure Jun2021 Popular

451 downloads

Download (pdf, 1.10 MB)

AXO-AAV-GM2 Trial Brochure Jun2021.pdf

pdf TSHA 101 Community Letter Popular

889 downloads

Download (pdf, 192 KB)

TSHA-101 Community Letter.pdf

pdf TSHA 101 Queens Trial Brochure Popular

704 downloads

Download (pdf, 423 KB)

TSHA-101 Queens Trial Brochure.pdf

pdf TSHA 101 Queens Trial Q&A Popular

683 downloads

Download (pdf, 183 KB)

TSHA-101 Queens Trial Q&A.pdf

pdf NTSAD Late Onset GM2 Listening Session Summary Popular

1402 downloads

Download (pdf, 60 KB)

NTSAD_Listening_Session_Summary.pdf

pdf Axovant GeneTherapies Announces U.S. FDA Clearance of IND for Registrational Study AXO AAV GM2 Popular

507 downloads

Download (pdf, 70 KB)

2020 AxovantGeneTherapies-Announces-FDA-Clearance-of-IND-for-Registrational-Study-AXO-AAV-GM2.pdf

Press Release - Axovant Gene Therapies Announces US FDA Clearance of IND for Registrational Study of AXO-AAV-GM2 Gene Therapy in Tay-Sachs and Sandhoff Diseases, allowing clinical trials in the United States.

pdf FAQs Venglustat Popular

1170 downloads

Download (pdf, 727 KB)

FAQs-Venglustat.pdf

pdf Axovant Open Letter to Patients and Advocacy Groups Popular

973 downloads

Download (pdf, 161 KB)

Axovant Open Letter to Patients and Advocacy Groups.pdf

pdf NTSAD 2016-2017 Request for Proposals Application and Guidelines Popular

1998 downloads

Download (pdf, 394 KB)

RFP Guidelines 2017.pdf

pdf Late Onset Medication Effects Study Presentation Popular

1216 downloads

Download (pdf, 973 KB)

LateOnset-Medication-Survey-062504-PPT.pdf

Medication Survey in Patients with LOTS
Kyle Anthony, BAKyle BA
Susan HattersSusan Hatters--Friedman, M.D.
Jose Americo Fernandes Filho, M.D.
Barbara E. Shapiro, M.D.,Ph.D.
University Hospitals of Cleveland University Cleveland
Case Western Reserve University School of Medicine Case Medicine

pdf Late Onset Medication Study (c.2004) Popular

3224 downloads

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LateOnset-Adverse-Effects-of-Medications-Neurology2006.pdf

Abstract—

The authors conducted a retrospective and brief prospective study of adverse effects of approximately 350 medications in 44 adults with late onset Tay–Sachs disease (LOTS). Some medications were relatively safe, whereas others, particularly haloperidol, risperidone, and chlorpromazine,
were associated with neurologic worsening.

pdf NTSAD 2015-2016 RFP Guidelines Popular

1539 downloads

Download (pdf, 471 KB)

RFP Guidelines 2015-16.pdf

2016 Request for Proposal announced.  Pre-applications due December 22.

NTSAD is soliciting proposals for innovative research projects that involve basic research, translational studies or clinical studies in the areas of neurodegenerative disorders affecting the CNS, including lysosomal storage disorders (particularly Tay-Sachs, GM-1, Sandhoff) and pediatric leukodystrophies (in particular Canavan disease).

 

pdf 2015 NTSAD Science Symposium & Workshop Agenda Popular

1412 downloads

Download (pdf, 629 KB)

NTSAD Scientific Symposium and Workshop agenda for April 16 2015.pdf

A schedule for the NTSAD Science Symposium & Workshop to be held April 16, 2015 in Reston, Virginia at the Hyatt Regency hotel. 

 

pdf Lysosomal Disease Network Fellowship Grant Popular

1289 downloads

Download (pdf, 99 KB)

LDN-FELLOWSHIP-APPLICATIONS-2015.pdf

The Lysosomal Disease Network accepting applications until Friday, February 6th, 5:00 PM (CST).

pdf 2014-2015 Request for Proposals Guidelines UPDATED Popular

1624 downloads

Download (pdf, 472 KB)

RFP Guidelines updated FINAL with updated due date.pdf

pdf NTSAD 2014-2015 RFP Guidelines Popular

1085 downloads

Download (pdf, 543 KB)

RFP Guidelines WEB.pdf

Updated guidelines can be found here with new deadlines. 

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