Results 171 to 180 of about 8,071 (187)
Some of the next articles are maybe not open access.

Patient reported outcomes in MPS IVA patients receiving enzyme replacement therapy

Molecular Genetics and Metabolism, 2019
Jacqueline Adam   +2 more
openaire   +1 more source

“Little people, big world”: Morquio syndrome type A (MPS IVA) in adult life

Molecular Genetics and Metabolism, 2020
Tatiane C. Roncato   +7 more
openaire   +1 more source

Uncovering mitochondrial disturbances in MPS IVA chondrocytes

Molecular Genetics and Metabolism
Andres F. Leal   +2 more
openaire   +1 more source

NICE recommends elosulfase-α for MPS-IVa

PharmacoEconomics & Outcomes News, 2015
openaire   +1 more source

55. A case report of Morquio syndrome (MPS IVA) with Morgagni diaphragmatic hernia

Molecular Genetics and Metabolism, 2010
Susan Hale   +6 more
openaire   +1 more source

Morquio A (MPS IVA) Syndrome

Mary C. Theroux   +3 more
openaire   +1 more source

Simultaneous measurement of lysosomal enzyme activities associated with MPS II, MPS IIIB, MPS IVA, MPS IVB, MPS VI, MPS VII, and CLN 2 disorders from a dried blood spot via LC-MS/MS

2018
Smith, Sara E   +5 more
openaire   +1 more source

Development of MPS IVA Syndrome Cellular Models Using Patient Specific Induced Pluripotent Stem Cells

Molecular Genetics and Metabolism, 2012
Andrzej Swistowski   +4 more
openaire   +1 more source

Crispr/Cas9-edited hematopoietic stem cells rescue MPS IVA fibroblasts phenotype

Molecular Genetics and Metabolism
Angelica Maria Herreno Pachon   +4 more
openaire   +1 more source

Insights from the Czech MPS IVA patient cohort, effect of therapy

Molecular Genetics and Metabolism
Robert Saho, Martin Magner
openaire   +1 more source
mucopolysaccharidosis iva
morquio a syndrome
medicine
mucopolysaccharidosis
enzyme replacement therapy
medicine general
morquio syndrome
mucopolysaccharidoses
galns
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