Canavan Research - NTSAD

2019 October Aspa poster Scott DW et al Canavan BBP 12 administration study

peg@pegdoyle.com (Peg Doyle) — Tue, 29 Oct 2019 12:23:18 -0400

A route of administration study of BBP-812, an AAV9-based gene therapy for the treatment of Canavan disease, in juvenile cynomolgus macaques

Scott DW, Rouse JL, Romero KB, Eclov R, Lewis TEW, Kapadia M, Mansfield G, and Beard CW
Aspa Therapeutics, BridgeBio, Raleigh, NC , USA

2019 October Aspa reports Scott DW et al Canavan BBP 12 administration study

peg@pegdoyle.com (Peg Doyle) — Tue, 29 Oct 2019 12:19:44 -0400

Aspa Therapeutics is pleased to share the news that a poster was presented on October 22, 2019 at the European Society of Gene and Cell Therapy meeting, describing an important development in gene therapy for Canavan disease.

Animal model Canavan TRAKA

Diana@ntsad.org (Diana) — Fri, 21 Sep 2018 11:28:56 -0400

Clinical Course Canavan BLEY

Diana@ntsad.org (Diana) — Fri, 21 Sep 2018 11:28:54 -0400

Canavan Natural History Study - MDBR_Bley

Diana@ntsad.org (Diana) — Mon, 15 Feb 2016 15:22:23 -0500

International Innovation - Dr. Gao

Diana@ntsad.org (Diana) — Thu, 11 Jun 2015 15:29:27 -0400

Genetics expert and microbiologist, Dr. Guangping Gao, details the experiences that led to him to devoting his scientific career to tackling Canavan disease, and illuminates the successes
he and his collaborators have achieved in this area.

Canavan Natural History - Informed Consent

Diana@ntsad.org (Diana) — Wed, 29 Apr 2015 13:59:57 -0400

Canavan Natural History - Questionnaire

Diana@ntsad.org (Diana) — Wed, 29 Apr 2015 13:58:56 -0400

Your participation in this study will help us better understand Canavan disease and why patients with the same disease often have a quite different course. Such knowledge is a prerequisite for evaluating new experimental therapies and assessing the effectiveness of palliative measures. Be sure to sign and send the informed consent as well which you can download here as well.

Canavan Natural History - Letter

Diana@ntsad.org (Diana) — Wed, 29 Apr 2015 13:54:49 -0400

Thanks to Team NTSAD, U Penn, and the Million Dollar Bike Ride a grant was made last year to Annette Bley, MD, University Hospital, Hamburg Eppendorf, Germany, for her proposal,

Quantitative description of the clinical course of Canavan disease. The study will be done in cooperation with Florian Eichler, MD, and others.

Read her letter of introduction and then download the questionnaire.

Making the White Matter Matters: Progress in Understanding Canavan’s Disease and Therapeutic Interventions Through Eight Decades

peg@pegdoyle.com (Peg Doyle) — Mon, 09 Mar 2015 09:28:33 -0400

Seemin S. Ahmed • Guangping Gao

Canavan Review Article

Canavan disease was first described in 1931, and this review traces the progress made in the past 8 decades to understand the disease by enumerating current hypotheses and ongoing palliative measures to alleviate patient symptoms in the context of the latest advances in the field.

SUMMARY Traka Canavan Lay Summary

(Anonymous) — Tue, 03 Jun 2014 11:16:19 -0400

A research summary on the Canavan disease.

Progress Report Traka 18 month report Canavan

(Anonymous) — Tue, 03 Jun 2014 11:16:18 -0400

A Research Project Summary (Feb 2012)

Progress Report Namboodiri Canavan tremor model

(Anonymous) — Tue, 03 Jun 2014 11:16:16 -0400

The progress report on the effectiveness of acetate supplementation on mice/rats.

Progress Report Gao Canavan gene therapy 02 2012

(Anonymous) — Tue, 03 Jun 2014 11:16:15 -0400

A progress report on the successful rescue of early fatality of Canavan mice by single IV injections of novel gene therapeutics.

Progress Report Gao FINAL

(Anonymous) — Tue, 03 Jun 2014 11:16:14 -0400

The final progress report, focusing on maximizing the efficiency of gene delivery to the CNS using the AAV9 capsid by
two aims.