1
Published 2018
Springer Berlin Heidelberg
Table of Contents: ...Assessment of the Effect of Once Daily Nitisinone Therapy on 24-h Urinary Metadrenalines and 5...

2
Published 2019
Springer Berlin Heidelberg
Table of Contents: ... -- Enzyme Replacement Therapy During Pregnancy in Fabry Patients -- Hyperornithinemia, Hyperammonemia...

3
Published 2016
Springer Berlin Heidelberg
Table of Contents: ... globotriaosylsphingosine levels after switching from agalsidase alfa to agalsidase beta as enzyme replacement therapy...

4
Published 2017
Springer Berlin Heidelberg
Table of Contents: ... -- Irreversibility of Symptoms with Biotin Therapy in an Adult with Profound Biotinidase Deficiency...

5
Published 2016
Springer Berlin Heidelberg
Table of Contents: ..., Complex MPS I -- Clinical Evolution After Enzyme Replacement Therapy in Twins with the Severe Form...

6
Published 2017
Springer Berlin Heidelberg
Table of Contents: ... Trifunctional Protein/Long-Chain 3-Hydroxy Acyl-CoA Dehydrogenase Deficiency -- N-Acetylcysteine Therapy...

7
Published 2017
Springer Berlin Heidelberg
Table of Contents: ... on Enzyme Replacement Therapy -- Newborn Screening Programmes in Europe, Arguments and Efforts Regarding...

8
Published 2019
Springer Berlin Heidelberg
Table of Contents: ... Synthetase Deficiency -- Enzyme Replacement Therapy in Pregnant Women with Fabry Disease: A Case Series...

9
Published 2018
Springer Berlin Heidelberg
Table of Contents: ... Cell Tumour of the Mandible: A Disease Association or Iatrogenic Complication of Therapy...

10
Published 2018
Springer Berlin Heidelberg
Table of Contents: ... Patients -- Triheptanoin: A Rescue Therapy for Cardiogenic Shock in Carnitine-acylcarnitine Translocase...

11
Published 2017
Springer Berlin Heidelberg
Table of Contents: ... Reactions to Enzyme Replacement Therapies -- Novel PEX3 Gene Mutations Resulting in a Moderate Zellweger...