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Peptide hormones / Hepatology / Glycogen storage disease type II / Mannose 6-phosphate / Insulin-like growth factor 1 / Insulin-like growth factor / BioMarin Pharmaceutical / Lysosomal storage disease / Alpha-glucosidase / Biology / Cell biology / Growth factors


Molecular Bases of Disease: Glycosylation-independent Lysosomal Targeting of Acid α-Glucosidase Enhances Muscle Glycogen Clearance in Pompe Mice John A. Maga, Jianghong Zhou, Ravi Kambampati, Susan Peng, Xu Wang, Richar
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Document Date: 2013-07-21 20:12:27

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File Size: 2,62 MB

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City

Gainesville / Bradford / Novato / Milwaukee / /

Company

BioMarin Pharmaceuticals Inc. / IgG / Pall / ZyStor Therapeutics Inc. / Adobe / Nikon / BMN / Millipore / Ligand / Genzyme Corp. / Synergy Software / Molecular Biology Inc. / rhGAA / PhysioGenix Inc. / Oncode-Med Inc. / GE Healthcare / BD Biosciences / BIAcore streptavidin SA / Bio-Rad / BMN 701 / Corning / generated using rhGAA / /

Currency

pence / /

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Event

FDA Phase / /

Facility

Stable CHO Cell Production / C terminus of GAA / Medical College of Wisconsin / University of Florida / N terminus / College of Medicine / N terminus of GAA / /

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IndustryTerm

saline solution / non-mammalian expression systems / therapeutics for other lysosomal storage diseases / therapies for the treatment of lysosomal storage diseases / enzyme replacement protocol / sensor chips / dose / treatment for patients with Pompe disease / proteolytic processing / low dose groups / cognate binding site / animals/ / therapy for Pompe disease / intracellular processing / biotinylation acceptor site / tissue site / tangential flow device / reconstituted commercial product / sensor chip / /

MedicalCondition

severe infant-onset Pompe disease / progressive muscle weakness / GAA deficiency / strain / muscle weakness / hypertropic cardiomyopathy / Pompe disease / dissociation / lysosomal storage disorder / cardiomyopathy / Disease / respiratory failure / Late-onset Pompe disease / chronic illness / lysosomal storage diseases / respiratory insufficiency / mucopolysaccharidosis VII / /

MedicalTreatment

enzyme replacement therapies / enzyme replacement therapy / /

Organization

Medical College of Wisconsin / Milwaukee / Department of Biochemistry / Administration of GILT-tagged BMN / FDA / §Powell Gene Therapy Center / College of Medicine / University of Florida / American Society for Biochemistry / /

Person

CHO LS / Nancy M. Dahms / Ravi Kambampati / Susan Peng / Peggy Tom / Barry J. Byrne / Richard N. Bohnsack / Angela Thomm / Jonathan H. LeBowitz / Sarah Golata / /

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Position

Author / representative / /

Product

Sodium acetate / glycine / Glycogen / Pompe / M6P / /

ProvinceOrState

New Jersey / Wisconsin / Florida / California / /

PublishedMedium

Molecular Biology / /

Technology

Antibodies / SA sensor chips / SA sensor chip / Gene Therapy / pharmacokinetics / DNA sequencing / 4-week enzyme replacement protocol / /

URL

http /

SocialTag