Medical research

New hope for Gaucher patients

What causes brain damage and inflammation in severe cases of Gaucher disease? Little is known about the events that lead to brain pathology in some forms of the disease, and there is currently no treatment available – a ...

Medical research

Fetal gene therapy prevents fatal neurodegenerative disease

A fatal neurodegenerative condition known as Gaucher disease can be prevented in mice following fetal gene therapy, finds a new study led by UCL, the KK Women's and Children's Hospital and National University Health System ...

Medical research

Complex mechanisms in Gaucher disease unravelled

Gaucher disease is a genetic disorder of lipid metabolism. Sphingosine, a compound as enigmatic as the sphinx, plays a key role in this metabolic disorder. Scientists from the Bonn research center caesar have identified some ...

Immunology

Nature study suggests new therapy for Gaucher disease

Scientists propose in Nature blocking a molecule that drives inflammation and organ damage in Gaucher and maybe other lysosomal storage diseases as a possible treatment with fewer risks and lower costs than current therapies.

Diseases, Conditions, Syndromes

Drug improves measures of genetic disease that affects liver, spleen

Among previously untreated adults with Gaucher disease type 1, a genetic disease in which there is improper metabolism due to a defect in an enzyme, treatment with the drug eliglustat resulted in significant improvements ...

Medications

Protalix signs supply deal with Brazilian govt

Shares of Protalix BioTherapeutics Inc. jumped in premarket trading Wednesday after the drug developer announced a deal that requires the Brazilian government to buy at least $280 million of the company's Gaucher disease ...

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