Examples of 'lysosomal storage disease' in a sentence

Meaning of "lysosomal storage disease"

Lysosomal storage disease: A group of rare inherited metabolic disorders characterized by an abnormal build-up of various toxic materials in the body's cells due to enzyme deficiencies. These diseases can cause a range of symptoms and complications depending on the specific disorder

How to use "lysosomal storage disease" in a sentence

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lysosomal storage disease
Galactosialidosis is a lysosomal storage disease.
Lysosomal storage disease enzymes.
If the individual has symptoms clinically associated with a certain lysosomal storage disease.
It is a lysosomal storage disease which is commonly listed in the family of leukodystrophies.
FD is considered a lysosomal storage disease.
The lysosomal storage disease aspartylglycosaminuria is caused by a deficiency in the AGA enzyme.
Deficiency in GCR activity results in a lysosomal storage disease referred to as Gaucher disease.
The lysosomal storage disease is Gaucher 's disease.
In some embodiments, the disease is a lysosomal storage disease.
Methods to treat the lysosomal storage disease can also include methods to treat Gaucher disease.
The therapeutic approach is directed to Niemann-Pick disease, a lysosomal storage disease.
It 's called a lysosomal storage disease or LSD.
In some embodiments, the human is suspected of suffering from a lysosomal storage disease.
The method of claim 6, wherein the lysosomal storage disease is Mucopolysaccharidosis.
Preferably, this site may be within a subject suffering from a lysosomal storage disease.

See also

The lysosomal storage disease may be any of the diseases identified in Table 1 above.
A deficiency of this enzyme causes the lysosomal storage disease alpha-mannosidosis.
Amicus sells a regulatory agency approved pharmacologic chaperone for Fabry disease-a lysosomal storage disease.
In another preferred embodiment, the lysosomal storage disease is Pompe Disease.
In some embodiments, the disease is a genetic disease, a cancer, a metabolic disease, or a lysosomal storage disease.
According to a preferred embodiment, the lysosomal storage disease is Gaucher disease.
Sanfilippo syndrome, or mucopolysaccharidosis III ( MPS-III ) is a rare autosomal recessive lysosomal storage disease.
In one embodiment, the lysosomal storage disease is Niemann-Pick disease.
In some embodiments, the human has been diagnosed with a lysosomal storage disease.
The method of 1, wherein the lysosomal storage disease is Niemann-Pick disease.
In some instances, the subject has or is at risk for having a lysosomal storage disease.
Preferably, the lysosomal storage disease is Gaucher 's disease.
The compound of Claim 1, for use in the treatment of a lysosomal storage disease.
In one embodiment, the lysosomal storage disease is Gaucher 's disease.
Key words, serum dolichol, aspartylglucosaminuria, neuronal ceroid-lipofuscinosis, lysosomal function, lysosomal storage disease.
The method of 1, wherein the lysosomal storage disease is Gaucher 's disease.
The compound for use according to Claim 4, wherein the lysosomal storage disease is Fabry.
In certain embodiments, the lysosomal storage disease has central nervous system ( CNS ) involvement.
In a certain disclosure the disease is a lysosomal storage disease ( LSD ).
Also most preferably, the lysosomal storage disease is Gaucher 's disease.
In certain embodiments, the disease is a lysosomal storage disease ( LSD ).
In certain embodiments, the lysosomal storage disease is Hunter Syndrome ( MPS II ).
In certain aspects the disease is a lysosomal storage disease ( LSD ).
In particularly preferred embodiments, the lysosomal storage disease is MPS VI.
The use or compound of claim 18, wherein the lysosomal storage disease is Fabry disease.

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Examples of using Lysosomal
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