disease

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Synonyms for disease

Synonyms for disease

a pathological condition of mind or body

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References in periodicals archive ?
Hematopoietic stem cell transplant for lysosomal storage diseases.
In lysosomal storage diseases like MPS I, enzymes needed to dissolve debris are missing, allowing debris to build up in cells until they malfunction.
Hepatomegaly and storage cells observed in both the bone marrow and liver biopsy with diffuse ascites strenghtened the suspicion of lysosomal storage disease.
CT}: Right now, the technology is becoming available and there is a whole push to get a number of lysosomal storage diseases as part of the newborn screening panel.
There is a pipeline of treatments that will soon become available for a variety of rare, lysosomal storage diseases," such as Niemann-Pick disease and Pompe's disease, Dr.
The status of hematopoietic stem cell transplantation in lysosomal storage disease.
Enzyme replacement therapy has been successful in treating one form of lysosomal storage disease called Gaucher disease.
Sanfilippo B, a lysosomal storage disease, is caused by deficiency in the enzyme alpha-N-acetyglucosaminidase (NAGLU), one of the four enzymes required for heparan sulfate degradation.
NPC1 is a lysosomal storage disease, characterized by too much cholesterol and other lipids in the cells lysosomes, which are sacs of enzymes that break apart proteins, fats and other materials for recycling.
The first-in-man clinical trial uses a single intravenous injection of AAV gene therapy for patients with MPS IIIA (Sanfilippo syndrome type A), a devastating lysosomal storage disease that affects every cell and organ resulting in neurocognitive decline, speech loss, loss of mobility, and premature death.
MPS IIIA or Sanfilippo A syndrome is an inherited systemic lysosomal storage disease with a significant central nervous system component.
Its Immune System Programming technology reprograms patient B cells to treat diseases, including MPS I, a rare genetic lysosomal storage disease expected to be its first clinical application.
Belonging to the group of mucopolysaccharidosis, Mucopolysaccharidosis IIIB (MPS IIIB) or Sanfilippo Syndrome Type B is a lysosomal storage disease.
As part of the collaboration Angiochem will initially create and develop an enzyme replacement therapy for a specified lysosomal storage disease while GSK will have the right to assume responsibility for development and commercialization of the resulting drug candidate.
Morquio A syndrome is a rare, autosomal recessive lysosomal storage disease caused by a deficiency in N-acetylgalactosamine-6-sulfate sulfatase (GALNS).
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