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GM1Gunner's Mate First Class (USN Rating)
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Release date- 13082019 - PHILADELPHIA - Passage Bio, a genetic medicines company developing AAV-delivered gene therapies for the treatment of rare monogenic central nervous system diseases, today announced the launch of a Natural History Study to evaluate patients with Type 1 and Type 2 GM1 gangliosidosis, a rare autosomal recessive genetic disorder that progressively destroys nerve cells in the brain and spinal cord.
The commercial uptake will likely be robust given the lack of approved therapies for patients with GM1 gangliosidosis, Skorney said.
is ongoing and a phase 1-2 clinical trial in GM1 Gangliosidosis is in preparation.
"With the introduction of our cutting-edge 0.8[eth]-m-pixel Samsung ISOCELL Bright GM1 and GD1 image sensors, we are committed to continue driving innovation in image sensor technologies."
The titer of ganglioside antibodies (GM1, GM1b, GD1a, and GaNac- GD1a) in the serum of AMAN patients is significantly increased.[1] The prodromal symptoms of GBS are diarrhea and upper respiratory tract infections.
In other children included in the study, MRS showed decreased NAA and decreased NAA/Cr ratio, though a common finding based on biochemical study/ enzyme analysis/ genetic correlation, they were diagnosed as NCL/ GM1 gangliosidosis/ idiopathic developmental delay.
Additionally, it is has been shown that LacCer and complex glycosphingolipids of cellular membranes such as Gb3 and GM1 are direct binding structures for bacteria and viruses (like Haemophilus influenza, Neisseria meningitidis, and Polyomavirus) [114, 115].
Both CT and LT are composed of enzymatically active A subunit which possesses ADP-ribosylating activity and pentameric B subunits which possesses monosialoganglioside (GM1) receptor-binding site.
To evaluate the effects of ganglioside GM1 treatment (Santa Cruz Biotechnology) on differentiation, hTSCs were cultured for 17 days in an osteogenic medium or 21 days in adipogenic medium supplemented with 1, 10, 50, and 100 [micro]M GM1.
At 2 years of age, comprehensive screening for metabolic disorders was completed including mannosidosis, fucosidosis, metachromatic leukodystrophy, Sandhoff disease, lysosomal storage diseases, GM1 gangliosidosis, Krabbe disease, and mucopolysaccharidosis (types 13 and 6).
GM1 gangliosidosis is an autosomal recessive storage disorder, which occurs in 1 per 100,000 to 200,000 newborns [1].