Tag: Lipid storage disorders

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Recent Related Articles to Lipid storage disorders

First-ever therapy for rare genetic disease in babies to save lives on the NHS - NHS England

Added to Collection on: 2023-11-27 20:29:27

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Lipid storage disorders
Rare diseases
Sebelipase alfa
Kanuma
Lysosomal acid lipase deficiency
National Health Service
Fabry disease
Wolman
Acid lipase disease
NHS England

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First treatment recommended for infants with Wolman Disease

Added to Collection on: 2023-11-27 20:27:39

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Lipid storage disorders
Rare diseases
Breakthrough therapy
Orphan drugs
Biopharmaceuticals
Sebelipase alfa
Lysosomal acid lipase deficiency
Kanuma
Enzyme replacement therapy
Wolman
National Institute for Health and Care Excellence
Inborn errors of metabolism

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Are Rare Brain Diseases the Next Commercial Frontier for Gene Therapy? - MedCity News

Added to Collection on: 2024-01-02 15:28:00

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Lipid storage disorders
Rare diseases
Gene delivery
Molecular genetics
Lysosomal storage diseases
Gene therapy
Tay–Sachs disease
Fabry disease
Gaucher's disease
Metachromatic leukodystrophy
Lysosomal storage disease
Viral vector

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Lysosomal Acid Lipase Deficiency Treatment Market High Growth - openPR.com

Added to Collection on: 2024-05-08 17:22:25

Tags for this article:

Lipid storage disorders
Management theory
Business process
Acid lipase disease
Lipase
Lysosomal acid lipase deficiency
Market analysis
Lysosome
Market segmentation
SWOT analysis
Strategic management
Lysosomal lipase
market insights

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FDA ad comm favors Zevra's Niemann-Pick drug

Added to Collection on: 2024-08-05 12:55:50

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Peptide hormones
Lipid storage disorders
Rare diseases
In Denmark
Semaglutide
GLP-1 receptor agonist
23andMe
Food and Drug Administration
Niemann?Pick disease, type C
Glucagon-like peptide-1
Niemann?Pick disease
Anne Wojcicki

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Cell and Gene Therapy: Becoming More Ordinary. Still Extraordinarily Expensive

Added to Collection on: 2024-09-03 20:58:44

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Rare diseases
Lipid storage disorders
Drug discovery
Gene therapy
Biotechnology
Semaglutide
CAR T cell
Clinical trial
Therapy
Niemann?Pick disease, type C
Niemann?Pick disease

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Krabbe Disease Treatment Markets, Companies, and Growth Opportunities 2024-2030

Added to Collection on: 2024-09-11 16:44:21

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Biotechnology
Rare diseases
Stem cells
Leukodystrophies
Lipid storage disorders
Krabbe disease
Stem-cell therapy
Personalized medicine
Gene therapy
Galactosylceramidase
Krabbe
Cell therapy
ResearchAndMarkets.com

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BridgeBio gets FDA RMAT designation for potential gene therapy

Added to Collection on: 2024-09-11 16:58:53

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Food and Drug Administration
Lipid storage disorders
Rare diseases
Pharmacy
Pharmaceuticals policy
Regenerative Medicine Advanced Therapy
Canavan disease
Gene therapy
Fast track
Therapy
BridgeBio Pharma

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Myrtelle's Canavan Disease Gene Therapy rAAV-Olig001-ASPA Decreases N ...

Added to Collection on: 2024-10-02 16:16:50

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Applied genetics
Food and Drug Administration
Rare diseases
Lipid storage disorders
Canavan disease
Leukodystrophies
Regenerative Medicine Advanced Therapy
Gene therapy
Aspartoacylase
Adeno-associated virus
Therapy
N-Acetylaspartic acid
Myrtelle Inc.

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Neuronal Ceroid-Lipofuscinoses Market 2032 |Pfizer, Spark - openPR.com

Added to Collection on: 2024-10-15 15:14:23

Tags for this article:

Lipid storage disorders
Rare diseases
Senescence
Neurodegenerative disorders
NCLS
Battenin
Infantile neuronal ceroid lipofuscinosis
Tripeptidyl peptidase I
Batten disease
Neuronal ceroid lipofuscinosis
Neurodegenerative disease
NCL
DelveInsight Business Research LLP

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