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  2. GM2 gangliosidoses - Wikipedia

    en.wikipedia.org/wiki/GM2_gangliosidoses

    A multinational clinical trial investigating N-Acetyl-L-Leucine for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff) began in 2019 [14] Recruitment is ongoing. IntraBio is also conducting parallel clinical trials with N-Acetyl-L-Leucine for the treatment of Niemann-Pick disease type C [15] and Ataxia-Telangiectasia. [16]

  3. GM1 gangliosidoses - Wikipedia

    en.wikipedia.org/wiki/GM1_gangliosidoses

    The GM1 gangliosidoses, usually shortened to GM1, are gangliosidoses caused by mutation in the GLB1 gene resulting in a deficiency of beta-galactosidase.The deficiency causes abnormal storage of acidic lipid materials in cells of the central and peripheral nervous systems, but particularly in the nerve cells, resulting in progressive neurodegeneration.

  4. Gangliosidosis - Wikipedia

    en.wikipedia.org/wiki/Gangliosidosis

    Gangliosidosis contains different types of lipid storage disorders [1] caused by the accumulation of lipids known as gangliosides. There are two distinct genetic causes of the disease. There are two distinct genetic causes of the disease.

  5. GM1 - Wikipedia

    en.wikipedia.org/wiki/GM1

    GM1 gangliosidosis are inherited disorders that progressively destroy neurons in the brain and spinal cord as GM1 accumulates. Without treatment, this results in developmental decline and muscle weakness, eventually leading to severe retardation and death.

  6. These Are the 4 Stages of Heart Failure - AOL

    www.aol.com/four-stages-heart-failure-143005999.html

    Stage A Defined as: At high risk for heart failure but without structural heart disease (defects in the heart from aging, injury, infection, or heart defect at birth), or symptoms of heart failure.

  7. Stem cell therapy to correct heart failure in children could ...

    www.aol.com/stem-cell-therapy-correct-heart...

    Doctors and specialists at the Murdoch Children's Research Institute in Melbourne, Australia, are studying and reprogramming the potential of the blood to treat heart failure in children.

  8. GM2-gangliosidosis, AB variant - Wikipedia

    en.wikipedia.org/wiki/GM2-gangliosidosis,_AB_variant

    Signs and symptoms of GM2-gangliosidosis, AB variant are identical with those of infantile Tay–Sachs disease, except that enzyme assay testing shows normal levels of hexosaminidase A. [2] Infantile Sandhoff disease has similar symptoms and prognosis, except that there is deficiency of both hexosaminidase A and hexosaminidase B. Infants with this disorder typically appear normal until the age ...

  9. Ganglioside - Wikipedia

    en.wikipedia.org/wiki/Ganglioside

    A summary showing the causes of sphingolipidosis, including gangliosidosis. Gangliosides are also involved in several diseases: Influenza, in which haemagglutinin of influenza virus exploits certain gangliosides to enter and infect the cells expressing them.

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