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JIMD Reports
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July 21, 2020
The Lysosomal Diseases Testing Laboratory: A review of the past 47 years
David A Wenger, Paola Luzi
JIMD Reports
|
July 21, 2020
Evaluation of 11 years of newborn screening for maple syrup urine disease in the Netherlands and a systematic review of the literature: Strategies for optimization
Kevin Stroek, Anita Boelen, Marelle J Bouva, et al.
JIMD Reports
|
March 31, 2015
The Kuvan(®) Adult Maternal Paediatric European Registry (KAMPER) Multinational Observational Study: Baseline and 1-Year Data in Phenylketonuria Patients Responsive to Sapropterin
Friedrich K Trefz, Ania C Muntau, Florian B Lagler, et al.
JIMD Reports
|
May 8, 2023
Paracetamol toxicity in classic homocystinuria: Effect of <i>N</i>-acetylcysteine on total homocysteine
Nour Elkhateeb, Sarah Hyde, Sarah L Hogg, et al.
JIMD Reports
|
May 8, 2023
The remarkable journey of one female individual with ornithine transcarbamylase deficiency diagnosed post-mortem
RaeLynn Forsyth, Ryan H Peretz, Angela Dempsey, et al.
JIMD Reports
|
May 8, 2023
Pregnancy, delivery, and postpartum period in infantile liver failure syndrome type 2 due to variants in <i>NBAS</i>
Bianca Peters, Felix Wiemers, Dominic Lenz, et al.
JIMD Reports
|
January 28, 2014
Mutational Spectrum of the CTNS Gene in Egyptian Patients with Nephropathic Cystinosis
Neveen A Soliman, Mohamed A Elmonem, Lambertus van den Heuvel, et al.
JIMD Reports
|
May 23, 2014
Laronidase replacement therapy and left ventricular function in mucopolysaccharidosis I
Haruhito Harada, Hiroshi Niiyama, Atsushi Katoh, et al.
JIMD Reports
|
September 14, 2022
Bone disease in early detected Gaucher Type I disease: A case report
Vincenza Gragnaniello, Alessandro P Burlina, Renzo Manara, et al.
JIMD Reports
|
September 14, 2022
Recurrent metabolic alkalosis following ketone body treatment of adult mitochondrial trifunctional protein deficiency: A case report
Nina N Stolwijk, Mirjam Langeveld, Bart A W Jacobs, et al.
Page
of 125
Search research articles
Search
Showing results (451-460 of 1,250) with videos related to
Sort By:
Page
of 125
JIMD Reports
|
July 21, 2020
The Lysosomal Diseases Testing Laboratory: A review of the past 47 years
David A Wenger, Paola Luzi
JIMD Reports
|
July 21, 2020
Evaluation of 11 years of newborn screening for maple syrup urine disease in the Netherlands and a systematic review of the literature: Strategies for optimization
Kevin Stroek, Anita Boelen, Marelle J Bouva, et al.
JIMD Reports
|
March 31, 2015
The Kuvan(®) Adult Maternal Paediatric European Registry (KAMPER) Multinational Observational Study: Baseline and 1-Year Data in Phenylketonuria Patients Responsive to Sapropterin
Friedrich K Trefz, Ania C Muntau, Florian B Lagler, et al.
JIMD Reports
|
May 8, 2023
Paracetamol toxicity in classic homocystinuria: Effect of <i>N</i>-acetylcysteine on total homocysteine
Nour Elkhateeb, Sarah Hyde, Sarah L Hogg, et al.
JIMD Reports
|
May 8, 2023
The remarkable journey of one female individual with ornithine transcarbamylase deficiency diagnosed post-mortem
RaeLynn Forsyth, Ryan H Peretz, Angela Dempsey, et al.
JIMD Reports
|
May 8, 2023
Pregnancy, delivery, and postpartum period in infantile liver failure syndrome type 2 due to variants in <i>NBAS</i>
Bianca Peters, Felix Wiemers, Dominic Lenz, et al.
JIMD Reports
|
January 28, 2014
Mutational Spectrum of the CTNS Gene in Egyptian Patients with Nephropathic Cystinosis
Neveen A Soliman, Mohamed A Elmonem, Lambertus van den Heuvel, et al.
JIMD Reports
|
May 23, 2014
Laronidase replacement therapy and left ventricular function in mucopolysaccharidosis I
Haruhito Harada, Hiroshi Niiyama, Atsushi Katoh, et al.
JIMD Reports
|
September 14, 2022
Bone disease in early detected Gaucher Type I disease: A case report
Vincenza Gragnaniello, Alessandro P Burlina, Renzo Manara, et al.
JIMD Reports
|
September 14, 2022
Recurrent metabolic alkalosis following ketone body treatment of adult mitochondrial trifunctional protein deficiency: A case report
Nina N Stolwijk, Mirjam Langeveld, Bart A W Jacobs, et al.
Page
of 125