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JIMD Reports
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March 11, 2020
Improvement of diagnostic yield in carbamoylphosphate synthetase 1 (<i>CPS1</i>) molecular genetic investigation by RNA sequencing
Jasmine Isler, Véronique Rüfenacht, Corinne Gemperle, et al.
JIMD Reports
|
March 17, 2021
Expression of tyrosine pathway enzymes in mice demonstrates that homogentisate 1,2-dioxygenase deficiency in the liver is responsible for homogentisic acid-derived ochronotic pigmentation
Peter J M Wilson, Lakshminarayan R Ranganath, George Bou-Gharios, et al.
JIMD Reports
|
March 17, 2021
Monitoring phenylalanine concentrations in the follow-up of phenylketonuria patients: An inventory of pre-analytical and analytical variation
Karlien L M Coene, Corrie Timmer, Susan M I Goorden, et al.
JIMD Reports
|
March 17, 2021
Enzyme replacement therapy interruption in mucopolysaccharidosis type IVA patients and its impact in different clinical outcomes
Juan Politei, Gloria Liliana Porras-Hurtado, Norberto Guelbert, et al.
JIMD Reports
|
January 21, 2021
Three-year follow up of using combination therapy with fresh-frozen plasma and iron chelation in a patient with acaeruloplasminemia
Andreas Tridimas, Godfrey T Gillett, Sally Pollard, et al.
JIMD Reports
|
January 21, 2021
Three successful pregnancies in a patient with glycogen storage disease type 0
Sarah C Grünert, Stefanie Rosenbaum-Fabian, Luciana Hannibal, et al.
JIMD Reports
|
January 21, 2021
Impact of trimethylaminuria on daily psychosocial functioning
Daniel Roddy, Philomena McCarthy, Darragh Nerney, et al.
JIMD Reports
|
January 21, 2021
Pregnancy outcome in women with Gaucher disease type 1 who had unplanned pregnancies during eliglustat clinical trials
Elena Lukina, Manisha Balwani, Nadia Belmatoug, et al.
JIMD Reports
|
November 12, 2021
Distal phalangeal erythema in an infant with biallelic <i>PDSS1</i> mutations: Expanding the phenotype of primary Coenzyme Q<sub>10</sub> deficiency
Marcello Bellusci, Maria Teresa García-Silva, Ana Martínez de Aragón, et al.
JIMD Reports
|
November 12, 2021
Divergent developmental trajectories in two siblings with neuropathic mucopolysaccharidosis type II (Hunter syndrome) receiving conventional and novel enzyme replacement therapies: A case report
Kazuyoshi Tomita, Shungo Okamoto, Toshiyuki Seto, et al.
Page
of 125
Search research articles
Search
Showing results (791-800 of 1,250) with videos related to
Sort By:
Page
of 125
JIMD Reports
|
March 11, 2020
Improvement of diagnostic yield in carbamoylphosphate synthetase 1 (<i>CPS1</i>) molecular genetic investigation by RNA sequencing
Jasmine Isler, Véronique Rüfenacht, Corinne Gemperle, et al.
JIMD Reports
|
March 17, 2021
Expression of tyrosine pathway enzymes in mice demonstrates that homogentisate 1,2-dioxygenase deficiency in the liver is responsible for homogentisic acid-derived ochronotic pigmentation
Peter J M Wilson, Lakshminarayan R Ranganath, George Bou-Gharios, et al.
JIMD Reports
|
March 17, 2021
Monitoring phenylalanine concentrations in the follow-up of phenylketonuria patients: An inventory of pre-analytical and analytical variation
Karlien L M Coene, Corrie Timmer, Susan M I Goorden, et al.
JIMD Reports
|
March 17, 2021
Enzyme replacement therapy interruption in mucopolysaccharidosis type IVA patients and its impact in different clinical outcomes
Juan Politei, Gloria Liliana Porras-Hurtado, Norberto Guelbert, et al.
JIMD Reports
|
January 21, 2021
Three-year follow up of using combination therapy with fresh-frozen plasma and iron chelation in a patient with acaeruloplasminemia
Andreas Tridimas, Godfrey T Gillett, Sally Pollard, et al.
JIMD Reports
|
January 21, 2021
Three successful pregnancies in a patient with glycogen storage disease type 0
Sarah C Grünert, Stefanie Rosenbaum-Fabian, Luciana Hannibal, et al.
JIMD Reports
|
January 21, 2021
Impact of trimethylaminuria on daily psychosocial functioning
Daniel Roddy, Philomena McCarthy, Darragh Nerney, et al.
JIMD Reports
|
January 21, 2021
Pregnancy outcome in women with Gaucher disease type 1 who had unplanned pregnancies during eliglustat clinical trials
Elena Lukina, Manisha Balwani, Nadia Belmatoug, et al.
JIMD Reports
|
November 12, 2021
Distal phalangeal erythema in an infant with biallelic <i>PDSS1</i> mutations: Expanding the phenotype of primary Coenzyme Q<sub>10</sub> deficiency
Marcello Bellusci, Maria Teresa García-Silva, Ana Martínez de Aragón, et al.
JIMD Reports
|
November 12, 2021
Divergent developmental trajectories in two siblings with neuropathic mucopolysaccharidosis type II (Hunter syndrome) receiving conventional and novel enzyme replacement therapies: A case report
Kazuyoshi Tomita, Shungo Okamoto, Toshiyuki Seto, et al.
Page
of 125