E-LIB Suche - Ergebnisse für: Lau, Heather

1

Long-Term Treatment of Gaucher Disease with Velaglucerase A..:

Deegan, Patrick ; Lau, Heather ; Elstein, Deborah...
Journal of Clinical Medicine. 13 (2024) 10 - p. 2782 , 2024

Link: https://doi.org/10.3390/..

2

Reply to Mistry et al. The Two Substrate Reduction Therapie..:

Hughes, Derralynn A. ; Deegan, Patrick ; Giraldo, Pilar...
Journal of Clinical Medicine. 12 (2023) 12 - p. 4017 , 2023

Link: https://doi.org/10.3390/..

3

Clinical characteristics and somatic burden of patients wit..:

Lau, Heather ; Harmatz, Paul ; Botha, Jaco..
Molecular Genetics and Metabolism Reports. 37 (2023) - p. 101005 , 2023

Link: https://doi.org/10.1016/..

4

Interim results of Transpher A, a multicenter, single-dose ..:

Flanigan, Kevin M. ; Smith, Nicholas ; Couce, Maria Luz...
Molecular Genetics and Metabolism. 138 (2023) 2 - p. 107101 , 2023

Link: https://doi.org/10.1016/..

5

Personal perspectives of international caregivers and a cli..:

Lopez, Antonio Gonzalez-Meneses ; Campbell, Ursula ; Guardia, Maria..
Molecular Genetics and Metabolism. 138 (2023) 2 - p. 107123 , 2023

Link: https://doi.org/10.1016/..

6

Efficacy and Safety of N-Acetyl-l-Leucine in Children and A..:

Martakis, Kyriakos ; Claassen, Jens ; Gascon-Bayari, Jordi...
Neurology. 100 (2023) 10 - p. , 2023

Link: https://doi.org/10.1212/..

7

The risk of hepatocellular carcinoma is markedly increased ..:

Basiri, Mohsen ; Ruan, Jiapeng ; Nair, Shiny...
Molecular Genetics and Metabolism. 138 (2023) 2 - p. 107022 , 2023

Link: https://doi.org/10.1016/..

8

First-in-human in vivo genome editing via AAV-zinc-finger n..:

Harmatz, Paul ; Prada, Carlos E. ; Burton, Barbara K....
Molecular Therapy. 30 (2022) 12 - p. 3587-3600 , 2022

Link: https://doi.org/10.1016/..

9

Vestronidase alfa for the treatment of mucopolysaccharidosi..:

Giugliani, Roberto ; Gonzalez-Meneses, Antonio ; Scarpa, Maurizio...
Molecular Genetics and Metabolism. 135 (2022) 2 - p. S47 , 2022

Link: https://doi.org/10.1016/..

10

UPDATED RESULTS FROM A NOVEL, LONGITUDINAL, MULTICENTER DIS..:

Giugliani, Roberto ; Gonzalez-Meneses, Antonio ; Scarpa, Maurizio...
Molecular Genetics and Metabolism. 135 (2022) 4 - p. 273-274 , 2022

Link: https://doi.org/10.1016/..

11

A natural history study of Sanfilippo syndrome type D (MPS ..:

Lau, Heather A. ; Mistry, Pramod ; Wood, Jill.
Molecular Genetics and Metabolism. 135 (2022) 2 - p. S71 , 2022

Link: https://doi.org/10.1016/..

12

Switching between Enzyme Replacement Therapies and Substrat..:

Hughes, Derralynn A. ; Deegan, Patrick ; Giraldo, Pilar...
Journal of Clinical Medicine. 11 (2022) 17 - p. 5158 , 2022

Link: https://doi.org/10.3390/..

13

Long-term efficacy and safety of vestronidase alfa enzyme r..:

Lau, Heather A. ; Viskochil, David ; Tanpaiboon, Pranoot...
Molecular Genetics and Metabolism. 136 (2022) 1 - p. 28-37 , 2022

Link: https://doi.org/10.1016/..

14

eP166: Vestronidase alfa for the treatment of Mucopolysacch..:

Guigliani, Roberto ; Gonzalez-Meneses Lopez, Antonio ; Scarpa, Maurizio...
Genetics in Medicine. 24 (2022) 3 - p. S101 , 2022

Link: https://doi.org/10.1016/..

15

Nonconvulsive Status Epilepticus - A Potentially Underrecog..:

Pauley, Rachel ; Dygert, Levi ; Nelson, Aaron.
Neurology. 98 (2022) 18_supplement - p. , 2022

Link: https://doi.org/10.1212/..