Danon Disease News and Research

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Danon disease (or glycogen storage disease Type IIb) is a lysosomal storage disease that was first characterized by Danon in 1981. Danon Disease is an X-linked dominant disorder that predominantly affects cardiac muscle. It affects both males and females, although males tend to see the majority of mental retardation and muscle weakness.

Researchers identify new way to treat Danon disease using gene therapy

Danon disease is a very rare, life-threatening condition where the fundamental biological process of removing and recycling proteins does not work.

18 Mar 2020

UC San Diego researchers awarded two grants for investigating stem cell-based therapies

The governing board of the California Institute for Regenerative Medicine unanimously approved this week two grants worth more than $2.2 million to University of California San Diego School of Medicine researchers investigating stem cell-based therapies for a rare genetic disorder that affects the heart and a chronic, progressive affliction of the lungs.

20 Jul 2018

Gene therapy may be effective method for treating Niemann-Pick disease, type C1

For the first time, National Institutes of Health researchers have demonstrated in mice that gene therapy may be the best method for correcting the single faulty gene that causes Niemann-Pick disease, type C1 (NPC1).

26 Oct 2016

UCSD scientists receive new CIRM grants to conduct stem cell-based research

Five scientists from the University of California, San Diego and its School of Medicine have been awarded almost $12 million in new grants from the California Institute for Regenerative Medicine (CIRM) to conduct stem cell-based research into regenerating spinal cord injuries, repairing gene mutations that cause amyotrophic lateral sclerosis and finding new drugs to treat heart failure and Alzheimer's disease.

28 May 2012

Recently identified genetic heart disorder often deadly for young patients

A study that included young patients with a recently recognized rare type of cardiomyopathy (a disorder of the heart muscle) linked to a genetic mutation finds that progression of this disease may be rapid and often results in early death, according to a study in the March 25 issue of JAMA.

24 Mar 2009