Canavan Disease

Common Name(s)

Canavan Disease, Spongy degeneration of central nervous system

Canavan disease is an inherited disorder that causes progressive damage to nerve cells in the brain. This disease is one of a group of genetic disorders called leukodystrophies. Leukodystrophies are characterized by degeneration of myelin, which is the fatty covering that insulates nerve fibers.  Canavan disease is caused by mutations in the ASPA gene and is inherited in an autosomal recessive pattern. While it occurs in people of all ethnic backgrounds, it is most common in people of Ashkenazi (eastern and central European) Jewish heritage, and among Saudi Arabians.
 
 

Advocacy and Support Organizations

 

Condition Specific Organizations

Following organizations serve the condition "Canavan Disease" for support, advocacy or research.

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Canavan Foundation, Inc.

The Foundation's mission is twofold: to help prevent Canavan disease and other Jewish genetic diseases through education about the availability of carrier screening; and to support research efforts to find effective therapies and a cure.

Last Updated: 15 Nov 2012

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Canavan Research Foundation

The Canavan Research Foundation is dedicated to eradicating neurodegenerative brain disease. Our research culminated in the world's first gene therapy for a brain disease in 1996, which spurred myelin growth in the brain of its young recipients. In the past few months, there has been a major breakthrough- through stem cell and metabolic treatment, the research team has found a cure to Canavan Disease, which will lead to a cure for Alzheimer's and other degenerative diseases. But funding is direly needed- your help has never been so important.

Last Updated: 15 Feb 2013

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Canavan Research Illinois

Canavan Research Illinois (also known as Canavan Disease Research) is a nonprofit corporation and 501(c)(3) public charity based in Illinois, established in April 2000 to meet a critical need to support medical research to treat, cure, and improve the quality of lives of all children battling Canavan disease, a rare fatal genetic neurological disorder. We further increase public awareness and network Canavan families.

Last Updated: 10 Jan 2013

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Center for Jewish Genetics

The Center is dedicated to gathering and disseminating knowledge about Jewish genetic disorders and hereditary cancers. Its mission is to educate and serve health care professionals, clergy and the Jewish community.

Last Updated: 26 Dec 2012

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CNS Foundation

CNS Foundation's Mission is Three-fold: - Expedite scientific research to find treatments and cures for the more than 14 million children living with some form of neurological disorder - Provide families and health care professionals with up-to-date information and resources on the latest discoveries in pediatric neurology - Educate the public, and public officials, on the critical importance of funding pediatric neurological disorder research

Last Updated: 12 Oct 2013

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Jacob's Cure

Jacob's Cure is dedicated to raising the funds necessary to support research aimed at treating and/or curing Canavan disease, a fatal, genetic, neurological disorder that affects children at birth.

Last Updated: 28 Nov 2012

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National Tay-Sachs & Allied Diseases Association, Inc.

The mission of the National-Tay Sachs & Allied Diseases Association is to lead the fight to treat and cure Tay-Sachs, Canavan and related genetic diseases and to support affected families and individuals in leading fuller lives.

Last Updated: 13 May 2014

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The Stennis Foundation

Raising awareness of the Leukodystrophies, and raising funds for Leukodystrophies research.

Last Updated: 14 Jan 2013

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General Support Organizations

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Advocacy and Support Organizations

 

Condition Specific Organizations

Following organizations serve the condition "Canavan Disease" for support, advocacy or research.

Logo
Canavan Foundation, Inc.

The Foundation's mission is twofold: to help prevent Canavan disease and other Jewish genetic diseases through education about the availability of carrier screening; and to support research efforts to find effective therapies and a cure.

http://www.canavanfoundation.org

Last Updated: 15 Nov 2012

View Details
Canavan Research Foundation

The Canavan Research Foundation is dedicated to eradicating neurodegenerative brain disease. Our research culminated in the world's first gene therapy for a brain disease in 1996, which spurred myelin growth in the brain of its young recipients. In the past few months, there has been a major breakthrough- through stem cell and metabolic treatment, the research team has found a cure to Canavan Disease, which will lead to a cure for Alzheimer's and other degenerative diseases. But funding is direly needed- your help has never been so important.

http://www.canavan.org

Last Updated: 15 Feb 2013

View Details
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Canavan Research Illinois

Canavan Research Illinois (also known as Canavan Disease Research) is a nonprofit corporation and 501(c)(3) public charity based in Illinois, established in April 2000 to meet a critical need to support medical research to treat, cure, and improve the quality of lives of all children battling Canavan disease, a rare fatal genetic neurological disorder. We further increase public awareness and network Canavan families.

http://www.canavanresearch.org

Last Updated: 10 Jan 2013

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Center for Jewish Genetics

The Center is dedicated to gathering and disseminating knowledge about Jewish genetic disorders and hereditary cancers. Its mission is to educate and serve health care professionals, clergy and the Jewish community.

http://www.jewishgenetics.org

Last Updated: 26 Dec 2012

View Details
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CNS Foundation

CNS Foundation's Mission is Three-fold: - Expedite scientific research to find treatments and cures for the more than 14 million children living with some form of neurological disorder - Provide families and health care professionals with up-to-date information and resources on the latest discoveries in pediatric neurology - Educate the public, and public officials, on the critical importance of funding pediatric neurological disorder research

http://www.cnsfoundation.org

Last Updated: 12 Oct 2013

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Jacob's Cure

Jacob's Cure is dedicated to raising the funds necessary to support research aimed at treating and/or curing Canavan disease, a fatal, genetic, neurological disorder that affects children at birth.

http://www.jacobscure.org

Last Updated: 28 Nov 2012

View Details
National Tay-Sachs & Allied Diseases Association, Inc.

The mission of the National-Tay Sachs & Allied Diseases Association is to lead the fight to treat and cure Tay-Sachs, Canavan and related genetic diseases and to support affected families and individuals in leading fuller lives.

http://www.ntsad.org

Last Updated: 13 May 2014

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The Stennis Foundation

Raising awareness of the Leukodystrophies, and raising funds for Leukodystrophies research.

http://www.stennisfoundation.org

Last Updated: 14 Jan 2013

View Details

 

General Support Organizations

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Scientific Literature

Articles from the PubMed Database

Research articles describe the outcome of a single study. They are the published results of original research.
The terms "Canavan Disease" returned 16 free, full-text research articles on human participants. First 3 results:

Long-term follow-up after gene therapy for canavan disease.
 

Author(s): Paola Leone, David Shera, Scott W J McPhee, Jeremy S Francis, Edwin H Kolodny, Larissa T Bilaniuk, Dah-Jyuu Wang, Mitra Assadi, Olga Goldfarb, H Warren Goldman, Andrew Freese, Deborah Young, Matthew J During, R Jude Samulski, Christopher G Janson

Journal: Sci Transl Med. 2012 Dec;4(165):165ra163.

 

Canavan disease is a hereditary leukodystrophy caused by mutations in the aspartoacylase gene (ASPA), leading to loss of enzyme activity and increased concentrations of the substrate N-acetyl-aspartate (NAA) in the brain. Accumulation of NAA results in spongiform degeneration of white ...

Last Updated: 20 Dec 2012

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Modification of aspartoacylase for potential use in enzyme replacement therapy for the treatment of Canavan disease.
 

Author(s): Stephen Zano, Radhika Malik, Sylvia Szucs, Reuben Matalon, Ronald E Viola

Journal: Mol. Genet. Metab.. 2011 Feb;102(2):176-80.

 

Canavan disease is a fatal neurological disease without any effective treatments to slow the relentless progress of this disorder. Enzyme replacement therapy has been used effectively to treat a number of metabolic disorders, but the presence of the blood-brain-barrier presents an ...

Last Updated: 18 Jan 2011

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Impact of gene patents and licensing practices on access to genetic testing and carrier screening for Tay-Sachs and Canavan disease.
 

Author(s): Alessandra Colaianni, Subhashini Chandrasekharan, Robert Cook-Deegan

Journal: Genet. Med.. 2010 Apr;12(4 Suppl):S5-S14.

 

Genetic testing for Tay-Sachs and Canavan disease is particularly important for Ashkenazi Jews, because both conditions are more frequent in that population. This comparative case study was possible because of different patenting and licensing practices. The role of DNA testing differs ...

Last Updated: 15 Apr 2010

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Reviews from the PubMed Database

Review articles summarize what is currently known about a disease. They discuss research previously published by others.
The terms "Canavan Disease" returned 0 free, full-text review articles on human participants.

 
 
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Clinical Trial Information This information is provided by ClinicalTrials.gov

Oral Glyceryl Triacetate (GTA) in Newborns With Canavan
 

Status: Recruiting

Condition Summary: Canavan Disease

 

Last Updated: 20 Jun 2011

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Efficacy Study of an Online Educational Module Before Carrier Genetic Screening in Persons of Ashkenazi Jewish Descent.
 

Status: Recruiting

Condition Summary: Tay Sachs Disease; Canavan Disease; Familial Dysautonomia

 

Last Updated: 24 Jul 2014

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