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Tay-Sachs, Canavan, GM1 and Sandhoff diseases

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Details: Last Updated: Monday, 18 November 2019 10:26

A collection of news from www.news-medical.net, an Internet resource.

News Feed: Tay-Sachs

Latest Tay Sachs Disease News and Research

Dual vector gene therapy for GM2 gangliosidosis shows encouraging results in clinical trial
Patients in a Phase I/II clinical trial conducted by UMass Chan Medical School of a dual vector gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases, exhibited a biochemical correction of the disease with minimal adverse reactions.
Protein identified as key to preventing fat build-up and related diseases
Accumulation of fat molecules is detrimental to the cell. Researchers from the Yong Loo Lin School of Medicine, National University of Singapore (NUS Medicine), have made a breakthrough in understanding how our cells manage to stay healthy by recycling important fat molecules.
Study identifies a potential treatment for Sandhoff and Tay-Sachs diseases
In a groundbreaking study, researchers at McMaster University have identified a potential treatment for Sandhoff and Tay-Sachs diseases—two rare, often fatal lysosomal storage disorders that cause progressive damage to nerve cells in the brain and spinal cord.

News Feed: Sandhoff

Latest Sandhoff Disease News and Research

Dual vector gene therapy for GM2 gangliosidosis shows encouraging results in clinical trial
Patients in a Phase I/II clinical trial conducted by UMass Chan Medical School of a dual vector gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases, exhibited a biochemical correction of the disease with minimal adverse reactions.
Targeted microglia replacement shows promise against fatal neurodegenerative conditions
Tay-Sachs and Sandhoff diseases, genetic disorders affecting the brain, have no effective treatment and are typically fatal within the first years of life.
Study identifies a potential treatment for Sandhoff and Tay-Sachs diseases
In a groundbreaking study, researchers at McMaster University have identified a potential treatment for Sandhoff and Tay-Sachs diseases—two rare, often fatal lysosomal storage disorders that cause progressive damage to nerve cells in the brain and spinal cord.

News Feed: Canavan Disease

Latest Canavan Disease (Leukodystrophies) News and Research

Human stem cells show promise against fatal children's diseases
Scientists have used human stem cells to dramatically improve the condition of mice with a neurological condition similar to a set of diseases in children that are invariably fatal, according to an article in the June issue of the journal Cell Stem Cell.

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News Feed: DNA Science

Geneticist Ricki Lewis blogs from the cutting edge of genomics, including genetic testing, stem cells, gene therapy and more.

An Avalanche of Advances in Human Genetics

For the first time in 35 years of writing and revising my textbook Human Genetics: Concepts and Applications, I’m happy to report…

The post An Avalanche of Advances in Human Genetics appeared first on DNA Science.
Cutting NIH Support of FlyBase Threatens Preclinical – and Ultimately Clinical – Research

The toppling of health care in the US has begun, and I fear will reverberate for decades to come. It is beginning…

The post Cutting NIH Support of FlyBase Threatens Preclinical – and Ultimately Clinical – Research appeared first on DNA Science.
Finally, a One-Time Treatment that May Slow the Course of Huntington’s Disease

It’s rare that clinical trial findings are as exciting as what biotech company UniQure has just announced. The company is based in…

The post Finally, a One-Time Treatment that May Slow the Course of Huntington’s Disease appeared first on DNA Science.

News Feed: Gangliosidosis-1

Latest Gangliosidosis News and Research

Dual vector gene therapy for GM2 gangliosidosis shows encouraging results in clinical trial
Patients in a Phase I/II clinical trial conducted by UMass Chan Medical School of a dual vector gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases, exhibited a biochemical correction of the disease with minimal adverse reactions.
ChatGPT: A diagnostic sidekick for doctors? Caution advised for non-professionals
Research evaluates the diagnostic accuracy of ChatGPT, an AI language model, in providing medical advice online. It concludes that ChatGPT can be a useful tool for assisting human doctors in diagnosing difficult cases, but non-professionals should use it with caution.
Gene therapy is the best stratagem to impact the course of GM2 gangliosidosis
Since its clinical description in 1881, Tay-Sachs disease has had almost totemic significance as a cruel and relentless genetic condition that destroys the developing brain of babies and young children.

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News Feeds

A collection of news from www.news-medical.net, an Internet resource.

News Feed: Tay-Sachs

News Feed: Sandhoff

News Feed: Canavan Disease

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News Feed: DNA Science

News Feed: Gangliosidosis-1

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