Tag: Enzyme replacement therapy

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Recent Related Articles to Enzyme replacement therapy

Avrobio pulls in $60M to push Fabry, Gaucher candidates through the clinic

Added to Collection on: 2018-01-31 20:00:00

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Health
Medicine
Biology
Rare diseases
Gene therapy
Lysosomal storage diseases
Lipid storage disorders
Fabry disease
Enzyme replacement therapy
Gaucher's disease
Glycogen storage disease type II
Cystinosis

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Capitalizing on a gene therapy boom, Avrobio locks in $60M

Added to Collection on: 2018-02-05 06:17:28

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Medicine
Biology
Biotechnology
Rare diseases
Gene therapy
Medical genetics
Lysosomal storage diseases
Lipid storage disorders
Fabry disease
Enzyme replacement therapy
Alpha-galactosidase
Geoff MacKay
CD34
Avrobio

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M6P Therapeutics Receives Six Rare Pediatric Disease Designations from the US FDA for ...

Added to Collection on: 2021-01-28 12:45:00

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Branches of biology
Biology
Clinical medicine
Lipid storage disorders
Rare diseases
Molecular biology
Lysosomal storage disease
Mannose 6-phosphate
Priority review
Gene therapy
Gaucher's disease
Enzyme replacement therapy
M6P Therapeutics

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Case Series Describes Hydroxychloroquine Use, Toxicity in Fabry Disease

Added to Collection on: 2021-02-11 15:45:00

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Clinical medicine
Branches of biology
Medicine
Lysosomal storage diseases
Rare diseases
Lipid storage disorders
Hydroxychloroquine
Quinolines
Fabry disease
Lysosomal storage disease
Alpha-galactosidase
Enzyme replacement therapy

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M6P Therapeutics Presents Promising Preclinical Data in Lysosomal Storage Disorders at the 17th ...

Added to Collection on: 2021-02-11 13:07:30

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Branches of biology
Inborn errors of metabolism
Enzymes
Glucocerebrosidase
Mannose 6-phosphate
Lysosomal storage disease
Lysosome
Gaucher's disease
I-cell disease
Beta-glucosidase
Lipid storage disorder
Enzyme replacement therapy

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Petition sent to Union Health Minister seeking sustainable treatment support for Gaucher Disease

Added to Collection on: 2024-10-19 15:26:29

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Rare diseases
Lipid storage disorders
Lysosomal storage disease
Gaucher's disease
Enzyme replacement therapy
Fabry disease
Gaucher
Roscoe Brady

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Spruce Axes 55% of Staff, Leaving Shoestring Crew To Focus on New Rare Disease Asset

Added to Collection on: 2025-04-28 20:59:42

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CLARIO
BioMarin Pharmaceutical
Enzyme replacement therapy
ERT
Coinbase
Spruce Biosciences

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Redefining Fabry Disease Care: Gene Therapy and Long-Acting ERT Prepare to Disrupt Status Quo

Added to Collection on: 2025-06-15 18:20:03

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Biotechnology
Rare diseases
Lipid storage disorders
Lysosomal storage diseases
Fabry disease
Molecular biology
GLA
Gene therapy
Protalix BioTherapeutics
Biopharmaceutical
Enzyme replacement therapy
Fabrazyme

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