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The US FDA has granted IntraBio a Rare Pediatric Disease Designation for N-Acetyl-Leucine for the treatment of GM2 Gangliosidosis. [ 12 ] Compassionate use studies in both Tay-Sachs and Sandhoff patients have demonstrated the positive clinical effects of treatment with N-Acetyl-Leucine for GM2 Gangliosidosis [ 13 ] These studies further ...
Heart disease is the most common underlying cause of arterial thromboembolism. It leads to the formation of blood clots in the heart, which leave it with the bloodstream and obstruct larger blood vessels, in cats mainly the aorta at the outlet of the two external iliac arteries. Arterial thromboembolism occurs suddenly and is very painful.
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.
Mutations in genes coding for these enzymes leads to the accumulation of partially broken down gangliosides in lysosomes, which results in a group of diseases called gangliosidosis. For example, the fatal Tay–Sachs disease arises as a genetic defect which leads to no functional hexosaminidase A produced, causing GM2 to accumulate in lysosomes.
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.
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.
The effect of treatment of blood pressure between 130/80 mmHg and 160/100 mmHg is less clear, with some reviews finding benefit [6] [16] [17] and others finding unclear benefit. [18] [19] [20] High blood pressure affects 33% of the population globally. [9] About half of all people with high blood pressure do not know that they have it. [9]
The presence of high blood pressure in diabetes is associated with a 4 fold increase in death chiefly from heart disease and strokes. [89] It has also been shown in recent epidemiological studies that variability of blood pressure, independent of mean blood pressure level, contributes to microvascular and macrovascular complications [ 90 ] in ...