Orphan designation: Recombinant human acid alpha-glucosidase, Treatment of glycogen storage disease type II (Pompe's disease)

Case Medical Research ◽

10.31525/cmr-1363a7c ◽

2019 ◽

Author(s):

Keyword(s):

Glycogen Storage Disease ◽

Storage Disease ◽

Glycogen Storage Disease Type ◽

Glycogen Storage ◽

Disease Type ◽

Type Ii ◽

Orphan Designation ◽

Pompe's Disease ◽

Alpha Glucosidase

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Orphan designation: miglustat, Treatment of glycogen storage disease type II (Pompe's disease)

Case Medical Research ◽

10.31525/cmr-ea52d0 ◽

2019 ◽

Author(s):

Keyword(s):

Glycogen Storage Disease ◽

Storage Disease ◽

Glycogen Storage Disease Type ◽

Glycogen Storage ◽

Disease Type ◽

Type Ii ◽

Orphan Designation ◽

Pompe’S Disease ◽

Pompe's Disease

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Orphan designation: Recombinant adeno-associated viral vector containing a bioengineered capsid serotype AAV-rh74 and a codon-optimised expression cassette to drive the expression of a secretable form of human acid alpha-glucosidase, Treatment of glycogen storage disease type II (Pompe's disease)

Case Medical Research ◽

10.31525/cmr-1ee8d09 ◽

2019 ◽

Author(s):

Keyword(s):

Glycogen Storage Disease ◽

Storage Disease ◽

Viral Vector ◽

Glycogen Storage Disease Type ◽

Glycogen Storage ◽

Disease Type ◽

Expression Cassette ◽

Type Ii ◽

Alpha Glucosidase

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Orphan designation: Recombinant adeno-associated viral vector containing human acid alfa-glucosidase-gene, Treatment of glycogen storage disease type II (Pompe's disease)

Case Medical Research ◽

10.31525/cmr-1584449 ◽

2019 ◽

Author(s):

Keyword(s):

Glycogen Storage Disease ◽

Storage Disease ◽

Viral Vector ◽

Glycogen Storage Disease Type ◽

Glycogen Storage ◽

Disease Type ◽

Type Ii ◽

Orphan Designation ◽

Gene Treatment ◽

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Orphan designation: miglustat, Treatment of glycogen storage disease type II (Pompe's disease)

Case Medical Research ◽

10.31525/cmr-ea7c24 ◽

2019 ◽

Author(s):

Keyword(s):

Glycogen Storage Disease ◽

Storage Disease ◽

Glycogen Storage Disease Type ◽

Glycogen Storage ◽

Disease Type ◽

Type Ii ◽

Orphan Designation ◽

Pompe’S Disease ◽

Pompe's Disease

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Increased Excretion of a Glucose-Containing Tetrasaccharide in the Urine of a Patient with Glycogen Storage Disease Type II (Pompe's Disease)

European Journal of Clinical Investigation ◽

10.1111/j.1365-2362.1974.tb02358.x ◽

1974 ◽

Vol 4 (6) ◽

pp. 429-433 ◽

Author(s):

P. Hallgren ◽

G. Hansson ◽

K. G. Henriksson ◽

A. Häger ◽

A. Lundblad ◽

...

Keyword(s):

Glycogen Storage Disease ◽

Storage Disease ◽

Glycogen Storage Disease Type ◽

Glycogen Storage ◽

Disease Type ◽

Type Ii ◽

Pompe’S Disease ◽

Pompe's Disease

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Packaging of an AAV vector encoding human acid α-glucosidase for gene therapy in glycogen storage disease type II with a modified hybrid adenovirus-AAV vector

Molecular Therapy ◽

10.1016/s1525-0016(03)00022-4 ◽

2003 ◽

Vol 7 (4) ◽

pp. 467-477 ◽

Author(s):

Baodong Sun ◽

Y.-T Chen ◽

Andrew Bird ◽

Fang Xu ◽

Yang-Xun Hou ◽

...

Keyword(s):

Gene Therapy ◽

Glycogen Storage Disease ◽

Storage Disease ◽

Glycogen Storage Disease Type ◽

Glycogen Storage ◽

Disease Type ◽

Type Ii ◽

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More on Bone Marrow Transplantation for Glycogen Storage Disease Type II (Pompé's Disease)

New England Journal of Medicine ◽

10.1056/nejm198611063151915 ◽

1986 ◽

Vol 315 (19) ◽

pp. 1229-1229

Keyword(s):

Bone Marrow ◽

Bone Marrow Transplantation ◽

Glycogen Storage Disease ◽

Marrow Transplantation ◽

Storage Disease ◽

Glycogen Storage Disease Type ◽

Glycogen Storage ◽

Disease Type ◽

Type Ii ◽

Pompe's Disease

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Recombinant human acid ??-glucosidase enzyme therapy for infantile glycogen storage disease type II: Results of a phase I/II clinical trial

Genetics in Medicine ◽

10.1097/00125817-200103000-00008 ◽

2001 ◽

Vol 3 (2) ◽

pp. 132-138 ◽

Author(s):

Andrea Amalfitano ◽

A. Resai Bengur ◽

Richard P. Morse ◽

Joseph M. Majure ◽

Laura E. Case ◽

...

Keyword(s):

Clinical Trial ◽

Phase I ◽

Glycogen Storage Disease ◽

Storage Disease ◽

Glycogen Storage Disease Type ◽

Glycogen Storage ◽

Disease Type ◽

Type Ii ◽

Enzyme Therapy ◽

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Long-Term Efficacy after [E1-, polymerase-] Adenovirus-Mediated Transfer of Human Acid-α-Glucosidase Gene into Glycogen Storage Disease Type II Knockout Mice

Human Gene Therapy ◽

10.1089/104303401750195917 ◽

2001 ◽

Vol 12 (8) ◽

pp. 955-965 ◽

Author(s):

E.Y. Ding ◽

B.L. Hodges ◽

H. Hu ◽

A.J. McVie-Wylie ◽

D. Serra ◽

...

Keyword(s):

Glycogen Storage Disease ◽

Knockout Mice ◽

Storage Disease ◽

Glycogen Storage Disease Type ◽

Glycogen Storage ◽

Disease Type ◽

Type Ii ◽

Term Efficacy ◽

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Galactosemia to Glycogen Storage Disease Type II (Pompe's Disease)

Neonatology ◽

10.1017/cbo9780511547270.021 ◽

2011 ◽

pp. 157-166

Keyword(s):

Glycogen Storage Disease ◽

Storage Disease ◽

Glycogen Storage Disease Type ◽

Glycogen Storage ◽

Disease Type ◽

Type Ii ◽

Pompe’S Disease ◽

Pompe's Disease

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