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Mucopolysaccharidosis IVA (MPS IVA, also known as Morquio A Syndrome) is a disease characterized by deficient activity of N-acetylgalactosamine-6-sulfatase (GALNS) causing excessive lysosomal storage of keratan sulfate (KS).
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Mucopolysaccharidosis IVA (MPS IVA, also known as Morquio A Syndrome) is a disorder characterized by deficient activity of N-acetylgalactosamine 6-sulfatase (GALNS) causing excessive lysosomal storage of keratan sulfate (KS).
unknown title 2009
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(GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome.
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We have also shown that GALNS is taken up into human Morquio chondrocytes in vitro and reaches the lysosome to clear keratan sulfate.
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We are finding that there are a number of Morquio patients out there, a large number in the clinic, worldwide, and many are here to be treated.
pfblogs.org: The Ad-Free Personal Finance Blogs Aggregator 2008
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It’s called Morquio Syndrome, which is a rare form of dwarfism.
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BioMarin announces update on BMN 110 Phase I/II trial for Morquio A Syndrome
THE MEDICAL NEWS 2010
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BioMarin's Clinical Trial Application for GALNS for Morquio A Syndrome Accepted by the MHRA
unknown title 2009
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announced today that its application for clinical trial authorization (CTA) for BMN 110 or N-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome, has been accepted by the United Kingdom Medicines and Healthcare Products Regulatory Agency (MHRA).
unknown title 2009
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Jean-Jacques Bienaime, Chief Executive Officer of BioMarin added, "Based on these data, we are more optimistic about the GALNS program as we continue to move closer to providing a treatment option for Morquio patients.
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