Impaired autophagy affects acid α-glucosidase processing and enzyme replacement therapy efficacy in late-onset glycogen storage disease type II

Replacement Therapy ◽

Storage Disease ◽

Late Onset ◽

Glycogen Storage ◽

Disease Type ◽

Type Ii ◽

Enzyme Replacement ◽

Therapy Efficacy

Exercise testing in late-onset glycogen storage disease type II patients undergoing enzyme replacement therapy

Neuromuscular Disorders ◽

10.1016/j.nmd.2012.10.017 ◽

2012 ◽

Vol 22 ◽

pp. S230-S234 ◽

Cited By ~ 8

Author(s):

Mauro Marzorati ◽

Simone Porcelli ◽

Giuseppe Bellistri ◽

Lucia Morandi ◽

Bruno Grassi

Keyword(s):

Replacement Therapy ◽

Exercise Testing ◽

Storage Disease ◽

Late Onset ◽

Glycogen Storage ◽

Disease Type ◽

Type Ii ◽

Pregnancy and associated events in women receiving enzyme replacement therapy for late-onset glycogen storage disease type II (Pompe disease)

Journal of Obstetrics and Gynaecology Research ◽

10.1111/jog.13055 ◽

2016 ◽

Vol 42 (10) ◽

pp. 1263-1271 ◽

Cited By ~ 7

Author(s):

Philippa J. Rohman ◽

Elaine Scott ◽

Linda Richfield ◽

Uma Ramaswami ◽

Derralynn A. Hughes

Keyword(s):

Replacement Therapy ◽

Pompe Disease ◽

Storage Disease ◽

Late Onset ◽

Glycogen Storage ◽

Disease Type ◽

Type Ii ◽

Glycogen storage disease type II (Pompe disease) - influence of enzyme replacement therapy in adults

European Journal of Neurology ◽

10.1111/j.1468-1331.2008.02377.x ◽

2009 ◽

Vol 16 (2) ◽

pp. 274-277 ◽

Cited By ~ 40

Author(s):

T. Merk ◽

T. Wibmer ◽

C. Schumann ◽

S. Krüger

Keyword(s):

Replacement Therapy ◽

Pompe Disease ◽

Storage Disease ◽

Glycogen Storage ◽

Disease Type ◽

Type Ii ◽

Effects of enzyme replacement therapy on five patients with advanced late-onset glycogen storage disease type II: a 2-year follow-up study

Journal of Inherited Metabolic Disease ◽

10.1007/s10545-011-9393-6 ◽

2011 ◽

Vol 35 (2) ◽

pp. 301-310 ◽

Cited By ~ 20

Author(s):

Yoshihiko Furusawa ◽

Madoka Mori-Yoshimura ◽

Toshiyuki Yamamoto ◽

Chikako Sakamoto ◽

Mizuki Wakita ◽

...

Keyword(s):

Storage Disease ◽

Late Onset ◽

Glycogen Storage ◽

Disease Type ◽

Type Ii ◽

Enzyme Replacement ◽

Follow Up Study

Enzyme replacement therapy in severe adult-onset glycogen storage disease type II

Advances in Therapy ◽

10.1007/s12325-008-0086-y ◽

2008 ◽

Vol 25 (8) ◽

pp. 820-829 ◽

Cited By ~ 14

Author(s):

Sabrina Ravaglia ◽

Cesare Danesino ◽

Anna Pichiecchio ◽

Alessandra Repetto ◽

Guy Umberto Poloni ◽

...

Keyword(s):

Replacement Therapy ◽

Storage Disease ◽

Glycogen Storage ◽

Disease Type ◽

Type Ii ◽

Adult Onset ◽

Progress in enzyme replacement therapy in glycogen storage disease type II

Therapeutic Advances in Neurological Disorders ◽

10.1177/1756285609103324 ◽

2009 ◽

Vol 2 (3) ◽

pp. 143-153 ◽

Cited By ~ 21

Author(s):

Corrado Angelini ◽

Claudio Semplicini ◽

Paola Tonin ◽

Massimiliano Filosto ◽

Elena Pegoraro ◽

...

Keyword(s):

Replacement Therapy ◽

Storage Disease ◽

Glycogen Storage ◽

Disease Type ◽

Type Ii ◽

Enzyme replacement therapy with alglucosidase alfa in 44 patients with late-onset glycogen storage disease type 2: 12-month results of an observational clinical trial

Journal of Neurology ◽

10.1007/s00415-009-5275-3 ◽

2009 ◽

Vol 257 (1) ◽

pp. 91-97 ◽

Cited By ~ 168

Author(s):

S. Strothotte ◽

N. Strigl-Pill ◽

B. Grunert ◽

C. Kornblum ◽

K. Eger ◽

...

Keyword(s):

Clinical Trial ◽

Storage Disease ◽

Late Onset ◽

Glycogen Storage ◽

Disease Type ◽

Enzyme Replacement ◽

Alglucosidase Alfa

Faculty Opinions recommendation of Alglucosidase alfa enzyme replacement therapy as a therapeutic approach for glycogen storage disease type III.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.717982893.793471770 ◽

2013 ◽

Author(s):

Janice Fletcher ◽

Shanti Balasubramaniam

Keyword(s):

Replacement Therapy ◽

Therapeutic Approach ◽

Storage Disease ◽

Glycogen Storage ◽

Disease Type ◽

Enzyme Replacement ◽

Alglucosidase Alfa

Alglucosidase alfa enzyme replacement therapy as a therapeutic approach for glycogen storage disease type III

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2012.12.002 ◽

2013 ◽

Vol 108 (2) ◽

pp. 145-147 ◽

Cited By ~ 12

Author(s):

Baodong Sun ◽

Keri Fredrickson ◽

Stephanie Austin ◽

Adviye A. Tolun ◽

Beth L. Thurberg ◽

...

Keyword(s):

Replacement Therapy ◽

Therapeutic Approach ◽

Storage Disease ◽

Glycogen Storage ◽

Disease Type ◽

Enzyme Replacement ◽

Alglucosidase Alfa

P4.51 Effects of enzyme replacement therapy in five patients with advanced late-onset glycogen storage disease type 2: a 2-year follow-up study

Neuromuscular Disorders ◽

10.1016/j.nmd.2010.07.247 ◽

2010 ◽

Vol 20 (9-10) ◽

pp. 674

Author(s):

Y.F. Furusawa ◽

M.M. Mori ◽

N.C. Chihara ◽

Y.M. Murata ◽

E.F. Endo ◽

...

Keyword(s):

Storage Disease ◽

Late Onset ◽

Glycogen Storage ◽

Disease Type ◽

Enzyme Replacement ◽

Follow Up Study