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

Latest News

Community News
Read NTSAD's latest e-newsletter! Sign up to receive next month's at the bottom of the home page. Read More
Request for Proposals 2023
Pre-applications due January 8, 2023. Read More
Day of Hope 2022
Create your own Day of Hope fundraising page! Read More

› ‹

News

News Feeds

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

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.
Known genes account for most undiagnosed cases of developmental disorders
Scientists have conducted the largest and most diverse study to date on how recessive genetic changes contribute to developmental disorders. They found that most undiagnosed cases that are due to recessive causes are linked to genes we already know about, and suggest a shift in research focus could improve diagnosis rates.

News Feed: Sandhoff

Latest Sandhoff Disease News and Research

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.
A new combination therapy for treating rare, fatal Sanfilippo syndrome in children
A study aiming to develop a new therapeutic technique could bring a revolution in our approach to treating rare, fatal Sanfilippo syndrome, a disorder that affects children as young as 2 years old and leads to childhood dementia and premature death.
New drug lures stem cells to damaged tissue, improves treatment efficacy
Scientists at Sanford Burnham Prebys Medical Discovery Institute have created a drug that can lure stem cells to damaged tissue and improve treatment efficacy--a scientific first and major advance for the field of regenerative medicine.

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.

Back Issues

Visit our library of eNews for copies of past newsletters, research updates, topics of the week and more

News Feed: DNA Science

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

Owning A Dog May Protect Against Eczema

About 100 million adults and 100 million children worldwide have the red, flaky, itchy skin of the most common form of eczema…

The post Owning A Dog May Protect Against Eczema appeared first on DNA Science.
A Trio of Novels Based on Rare Genetic Disease: Middlesex, The Covenant of Water, and The Sirens

I love when fiction unfurls a compelling tale whose protagonist has an ultrarare genetic disease. My most recent favorite is The Sirens…

The post A Trio of Novels Based on Rare Genetic Disease: Middlesex, The Covenant of Water, and The Sirens appeared first on DNA Science.
CRISPR Gene Editing Outpaces Gene Therapy: The Groundbreaking Case of KJ Muldoon

Nine-month-old KJ Muldoon made global headlines following a report in The New England Journal of Medicine, from Kiran Musunuru and Becca Ahrens-Nicklas…

The post CRISPR Gene Editing Outpaces Gene Therapy: The Groundbreaking Case of KJ Muldoon appeared first on DNA Science.

News Feed: Gangliosidosis-1

Latest Gangliosidosis News and Research

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.
New gene therapy shows promising results for treating neurodegenerative disorders
A new gene therapy approach designed to replace the enzyme that is deficient in patients with the inherited neurodegenerative disorders Tay-Sachs and Sandhoff diseases successfully delivered the therapeutic gene to the brains of treated mice, restored enzyme function, and extended survival by about 2.5-fold.

Latest News

Community News

Request for Proposals 2023

Day of Hope 2022

News

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

Back Issues

News Feed: DNA Science

News Feed: Gangliosidosis-1

NTSAD

2001 Beacon Street
Suite 204

Boston, MA 02135

(617) 277-4463

Follow NTSAD

Twitter

Facebook

YouTube

LinkedIn

Ask us how you can join the fight.

info@ntsad.org

Login