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2 research outputs found

Splice modulating antisense oligonucleotides restore some acid-alpha-glucosidase activity in cells derived from patients with late-onset Pompe disease

Author: A Amantana
AJ Bergsma
AT van der Ploeg
AWGoMoP Disease
B Schoser
BK Smith
C Betts
C Mitrpant
CA Schneider
CE Bekircan-Kurt
CF Boerkoel
DP Rastall
DP Rastall
E Goina
E van der Wal
E van der Wal
EJ Cupler
EW Ottesen
F Piva
G Limongelli
G McClorey
G McClorey
G Ronzitti
H Zhou
HM Moulton
J Hsu
J Kim
JR Mendell
JR Mendell
K Umapathysivam
KM Stepien
L Cao
L Chen
L Lattanzi
LDM Pena
M Chillon
M Kinali
M Kroos
M Mehler
ME McCready
MG Ausems
MI Nilsson
ML Huie
MM Hermans
MS Falzarano
MT Ganger
N Raben
N Raben
N Subramanian
N Zhong
PR Joshi
Q Zhou
QL Wang
RA Spritz
RE Palmer
RM Hudziak
SF Altschul
SM Hammond
SO Han
TM Bunnell
V Arechavala-Gomeza
Publication venue: 'Springer Science and Business Media LLC'
Publication date
Field of study

Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients

Author: A Lucia
A Renand
A Wroblewska
AC Nascimbeni
AF Santos
AH El-Gharbawy
AT Palermo
AT van der Ploeg
AT van der Ploeg
AT van der Ploeg
AWGoMoP Disease
B Knoechel
B Sun
CA Akdis
D Orlikowski
DP Germain
E Martinuzzi
EM Moran
F Dazzi
F Meiler
F Mingozzi
G Hu
G Shankar
GE Linthorst
GM Bartelds
H Galjaard
J Oldenburg
JM de Vries
JR Mendell
JS Park
JW Teener
KM Flanigan
M Banati
M Keiserman
M Kroos
M Nicolino
MJ Allen
NJ Mendelsohn
NJ Singh
P Dickson
PA Blair
PC Moorehead
PS Kishnani
PS Kishnani
R Kakavanos
RC Aalberse
S Nayak
S Nayak
SA Montalvao
SE Lipinski
SF Whelan
SG Banugaria
SG Banugaria
SN Prater
SO Han
SS Fakharzadeh
TT Patel
W van de Veen
YH Messinger
Publication venue: 'Springer Science and Business Media LLC'
Publication date
Field of study

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