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Latest enzyme replacement therapy Stories

2014-07-07 16:24:20

- MPS IV A patients and families urge swift, publicly funded access to life-altering drug VIMIZIM(TM) - VANCOUVER, July 7, 2014 /CNW/ - The Canadian Society for Mucopolysaccharide & Related Diseases Inc. (Canadian MPS Society) is thrilled by Health Canada's recent approval of VIMIZIM(TM) (elosulfase alfa), the first and only pharmaceutical treatment option available for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Mucopolysaccharidosis IVA (MPS...

2014-01-20 10:36:30

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 bleak outlook for sufferers and their families. Now, scientists at the Weizmann Institute of Science have discovered a new cellular pathway implicated in Gaucher disease. Their findings, published today in Nature Medicine, may offer a new therapeutic target for the...

2013-10-03 09:29:44

A genetic condition that attacks multiple organs and usually results in fatal heart problems can be detected using a new MRI technique that was developed at the University of Alberta. The discovery of this new diagnostic tool has resulted in updated clinical guidelines for the diagnosis and treatment of Fabry disease in Canada. Faculty of Medicine & Dentistry researchers Gavin Oudit and Richard Thompson worked with Faculty colleagues Kelvin Chow and Alicia Chan on the discovery, as...

2012-01-09 11:29:34

A team of scientists, led by researchers at the University of California, San Diego School of Medicine and Zacharon Pharmaceuticals, have developed a simple, reliable test for identifying biomarkers for mucopolysaccharidoses (MPS), a group of inherited metabolic disorders that are currently diagnosed in patients only after symptoms have become serious and the damage possibly irreversible. The findings will be published online January 8 in the journal Nature Chemical Biology. MPS is...

2012-01-06 10:31:44

Kids with Pompe disease fail because of a missing enzyme, GAA, that leads to dangerous sugar build-up, which affects muscles and movement. An enzyme replacement treatment pioneered at Duke University has saved many lives, but some children with Pompe disease produce an immune reaction that blocks the benefits of the life-saving enzyme treatment. To date there has been no success in eliminating or suppressing this immune response. Now research led by Duke University Medical Center, with...


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pungle
  • To take pains; labor assiduously with little progress.
This word comes from the Spanish 'pongale,' put it.
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