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Molecular Genetics and Metabolism
|
May 27, 2008
Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase
Paul Harmatz, Roberto Giugliani, Ida Vanessa D Schwartz, et al.
Frontiers in Immunology
|
May 1, 2026
Beyond the interferon score: neurofilament light chain and glial fibrillary acidic protein capture immune-mediated neuroinjury and response to JAK inhibition in Aicardi-Goutières syndrome
Lisa Wege, Christian Klemann, Sandy Siegert, et al.
Archives of Neurology
|
May 16, 2007
Hereditary spastic paraplegia 3A associated with axonal neuropathy
Neviana Ivanova, Kristl G Claeys, Tine Deconinck, et al.
Journal of Pediatric Rehabilitation Medicine
|
July 17, 2010
Enzyme replacement therapy for mucopolysaccharidosis VI: Growth and pubertal development in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase
Celeste Decker, Zi-Fan Yu, Roberto Giugliani, et al.
Journal of Inherited Metabolic Disease
|
February 9, 2010
Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase
Paul Harmatz, Zi-Fan Yu, Roberto Giugliani, et al.
Journal of Medical Genetics
|
August 10, 2014
The clinical significance of small copy number variants in neurodevelopmental disorders
Reza Asadollahi, Beatrice Oneda, Pascal Joset, et al.
Parkinsonism & Related Disorders
|
December 10, 2021
WARS2 mutations cause dopa-responsive early-onset parkinsonism and progressive myoclonus ataxia
Matej Skorvanek, Irena Rektorova, Wim Mandemakers, et al.
Frontiers in Genetics
|
April 29, 2015
Clinical, biochemical, and genetic spectrum of seven patients with NFU1 deficiency
Uwe Ahting, Johannes A Mayr, Arnaud V Vanlander, et al.
Nature Communications
|
March 12, 2016
CCC- and WASH-mediated endosomal sorting of LDLR is required for normal clearance of circulating LDL
Paulina Bartuzi, Daniel D Billadeau, Robert Favier, et al.
Journal of Medical Genetics
|
April 14, 2012
Molecular diagnosis in mitochondrial complex I deficiency using exome sequencing
Tobias B Haack, Birgit Haberberger, Eva-Maria Frisch, et al.
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of 12
Search research articles
Search
Showing results (81-90 of 113) with videos related to
Sort By:
Page
of 12
Molecular Genetics and Metabolism
|
May 27, 2008
Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase
Paul Harmatz, Roberto Giugliani, Ida Vanessa D Schwartz, et al.
Frontiers in Immunology
|
May 1, 2026
Beyond the interferon score: neurofilament light chain and glial fibrillary acidic protein capture immune-mediated neuroinjury and response to JAK inhibition in Aicardi-Goutières syndrome
Lisa Wege, Christian Klemann, Sandy Siegert, et al.
Archives of Neurology
|
May 16, 2007
Hereditary spastic paraplegia 3A associated with axonal neuropathy
Neviana Ivanova, Kristl G Claeys, Tine Deconinck, et al.
Journal of Pediatric Rehabilitation Medicine
|
July 17, 2010
Enzyme replacement therapy for mucopolysaccharidosis VI: Growth and pubertal development in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase
Celeste Decker, Zi-Fan Yu, Roberto Giugliani, et al.
Journal of Inherited Metabolic Disease
|
February 9, 2010
Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase
Paul Harmatz, Zi-Fan Yu, Roberto Giugliani, et al.
Journal of Medical Genetics
|
August 10, 2014
The clinical significance of small copy number variants in neurodevelopmental disorders
Reza Asadollahi, Beatrice Oneda, Pascal Joset, et al.
Parkinsonism & Related Disorders
|
December 10, 2021
WARS2 mutations cause dopa-responsive early-onset parkinsonism and progressive myoclonus ataxia
Matej Skorvanek, Irena Rektorova, Wim Mandemakers, et al.
Frontiers in Genetics
|
April 29, 2015
Clinical, biochemical, and genetic spectrum of seven patients with NFU1 deficiency
Uwe Ahting, Johannes A Mayr, Arnaud V Vanlander, et al.
Nature Communications
|
March 12, 2016
CCC- and WASH-mediated endosomal sorting of LDLR is required for normal clearance of circulating LDL
Paulina Bartuzi, Daniel D Billadeau, Robert Favier, et al.
Journal of Medical Genetics
|
April 14, 2012
Molecular diagnosis in mitochondrial complex I deficiency using exome sequencing
Tobias B Haack, Birgit Haberberger, Eva-Maria Frisch, et al.
Page
of 12