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Journal of Clinical Medicine
|
April 14, 2026
Improvement of Bone Mineral Density in Patients with Type 1 Gaucher Disease Treated with Velaglucerase Alfa: Results from Clinical Studies
Ari Zimran, Jaco Botha, Richard Eastell, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
August 4, 2017
Diagnosis and treatment of tyrosinemia type I: a US and Canadian consensus group review and recommendations
Jeffrey M Chinsky, Rani Singh, Can Ficicioglu, et al.
International Journal of Neonatal Screening
|
November 18, 2020
Newborn Screening for Pompe Disease: Pennsylvania Experience
Can Ficicioglu, Rebecca C Ahrens-Nicklas, Joshua Barch, et al.
Molecular Genetics and Metabolism
|
January 23, 2009
A Delphi clinical practice protocol for the management of very long chain acyl-CoA dehydrogenase deficiency
Georgianne L Arnold, Johan Van Hove, Debra Freedenberg, et al.
Molecular Genetics and Metabolism
|
June 6, 2008
Complex management of a patient with a contiguous Xp11.4 gene deletion involving ornithine transcarbamylase: a role for detailed molecular analysis in complex presentations of classical diseases
Matthew A Deardorff, Himabindu Gaddipati, Paige Kaplan, et al.
Molecular Genetics and Metabolism
|
April 3, 2022
The diagnosis and management of Gaucher disease in pediatric patients: Where do we go from here?
Neal J Weinreb, Ozlem Goker-Alpan, Priya S Kishnani, et al.
International Journal of Neonatal Screening
|
April 25, 2022
Newborn Screening for X-Linked Adrenoleukodystrophy: Review of Data and Outcomes in Pennsylvania
Jessica R C Priestley, Laura A Adang, Sarah Drewes Williams, et al.
Journal of Inherited Metabolic Disease
|
March 4, 2024
Efficacy and safety of sapropterin before and during pregnancy: Final analysis of the Kuvan® Adult Maternal Paediatric European Registry (KAMPER) maternal and Phenylketonuria Developmental Outcomes and Safety (PKUDOS) PKU-MOMs sub-registries
François Feillet, Can Ficicioglu, Florian B Lagler, et al.
Molecular Genetics and Metabolism
|
September 16, 2018
Pharmacokinetics of glycerol phenylbutyrate in pediatric patients 2 months to 2 years of age with urea cycle disorders
Susan A Berry, Jerry Vockley, Alexander A Vinks, et al.
Metabolic Brain Disease
|
February 14, 2018
Treatment outcome of creatine transporter deficiency: international retrospective cohort study
Theodora U J Bruun, Sarah Sidky, Anabela O Bandeira, et al.
Page
of 11
Search research articles
Search
Showing results (71-80 of 110) with videos related to
Sort By:
Page
of 11
Journal of Clinical Medicine
|
April 14, 2026
Improvement of Bone Mineral Density in Patients with Type 1 Gaucher Disease Treated with Velaglucerase Alfa: Results from Clinical Studies
Ari Zimran, Jaco Botha, Richard Eastell, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
August 4, 2017
Diagnosis and treatment of tyrosinemia type I: a US and Canadian consensus group review and recommendations
Jeffrey M Chinsky, Rani Singh, Can Ficicioglu, et al.
International Journal of Neonatal Screening
|
November 18, 2020
Newborn Screening for Pompe Disease: Pennsylvania Experience
Can Ficicioglu, Rebecca C Ahrens-Nicklas, Joshua Barch, et al.
Molecular Genetics and Metabolism
|
January 23, 2009
A Delphi clinical practice protocol for the management of very long chain acyl-CoA dehydrogenase deficiency
Georgianne L Arnold, Johan Van Hove, Debra Freedenberg, et al.
Molecular Genetics and Metabolism
|
June 6, 2008
Complex management of a patient with a contiguous Xp11.4 gene deletion involving ornithine transcarbamylase: a role for detailed molecular analysis in complex presentations of classical diseases
Matthew A Deardorff, Himabindu Gaddipati, Paige Kaplan, et al.
Molecular Genetics and Metabolism
|
April 3, 2022
The diagnosis and management of Gaucher disease in pediatric patients: Where do we go from here?
Neal J Weinreb, Ozlem Goker-Alpan, Priya S Kishnani, et al.
International Journal of Neonatal Screening
|
April 25, 2022
Newborn Screening for X-Linked Adrenoleukodystrophy: Review of Data and Outcomes in Pennsylvania
Jessica R C Priestley, Laura A Adang, Sarah Drewes Williams, et al.
Journal of Inherited Metabolic Disease
|
March 4, 2024
Efficacy and safety of sapropterin before and during pregnancy: Final analysis of the Kuvan® Adult Maternal Paediatric European Registry (KAMPER) maternal and Phenylketonuria Developmental Outcomes and Safety (PKUDOS) PKU-MOMs sub-registries
François Feillet, Can Ficicioglu, Florian B Lagler, et al.
Molecular Genetics and Metabolism
|
September 16, 2018
Pharmacokinetics of glycerol phenylbutyrate in pediatric patients 2 months to 2 years of age with urea cycle disorders
Susan A Berry, Jerry Vockley, Alexander A Vinks, et al.
Metabolic Brain Disease
|
February 14, 2018
Treatment outcome of creatine transporter deficiency: international retrospective cohort study
Theodora U J Bruun, Sarah Sidky, Anabela O Bandeira, et al.
Page
of 11