Tag: Orphan drug

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Recent Related Articles to Orphan drug

European Commission grants marketing authorization for acanthamoeba keratitis treatment

Added to Collection on: 2024-09-02 17:41:06

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Discosea
Acanthamoeba keratitis
Polyhexanide
Acanthamoeba
Keratitis
Contact lens
Orphan drug
Corneal transplantation
European Medicines Agency

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FDA Grants Breakthrough Therapy Designation to Plozasiran for FCS - HCPLive

Added to Collection on: 2024-09-12 14:36:28

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Lipid metabolism disorders
Rare diseases
Apolipoprotein C-III
Apolipoproteins
Lipoprotein lipase deficiency
Breakthrough therapy
Hypertriglyceridemia
Chylomicron
Orphan drug
Hyperlipidemia
Fast track
Arrowhead Pharmaceuticals Inc.

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Tenaya Therapeutics, Inc. SEC 10-Q Report - TradingView

Added to Collection on: 2024-11-06 22:22:23

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Drug discovery
Bioethics
Gene therapy
Clinical trial
Orphan drug
Tenaya Therapeutics Inc.

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Intellia Therapeutics Announces FDA Regenerative Medicine Advanced Therapy (RMAT ...

Added to Collection on: 2024-11-25 17:33:34

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Amyloidosis
Neurological disorders
Pharmaceuticals policy
Intellia Therapeutics
Protein folding
Familial amyloid polyneuropathy
Transthyretin
Regenerative Medicine Advanced Therapy
Orphan drug
Wild-type transthyretin amyloid
Alnylam Pharmaceuticals
CRISPR
Intellia Therapeutics Inc

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10 biotech companies to watch in 2025 - Labiotech.eu

Added to Collection on: 2024-12-16 14:29:24

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Biotechnology
Applied genetics
Bioethics
Gene therapy
Molecular biology
Orphan drug
PD-1 and PD-L1 inhibitors
Vertex Pharmaceuticals

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Cour Pharmaceuticals' PBC Treatment Gets FDA Orphan Drug Designation - PharmTech

Added to Collection on: 2025-01-09 19:27:30

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Hepatology
Cholestasis
Primary biliary cholangitis
Bile acid
Liver disease
Pyruvate dehydrogenase
Liver
Ascending cholangitis
Bile
Orphan drug
Ursodeoxycholic acid
Obeticholic acid
COUR Pharmaceuticals

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Rigel Announces R289 Granted Orphan Drug Designation by the FDA for MDS

Added to Collection on: 2025-01-09 15:36:29

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Food and Drug Administration
Pharmaceuticals policy
Drug discovery
Orphan drug
Fast track
Orphan Drug Act
Rigel Pharmaceuticals, Inc

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Orphan Drugs Strategic Business Report 2024-2030: - GlobeNewswire

Added to Collection on: 2025-01-22 10:59:13

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Pharmaceuticals policy
Rare diseases
Biotechnology law
Drug discovery
Orphan drug
Pharmaceutical industry
Rare disease
Medication
BioMarin Pharmaceutical
AstraZeneca
Vertex Pharmaceuticals
Sanofi

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Goodwin's 2025 Rare Disease Symposium: Momentum Builds for Addressing Critical ...

Added to Collection on: 2025-02-26 18:00:24

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Food and Drug Administration
Priority review
Rare disease
Orphan drug
National Organization for Rare Disorders
Patient advocacy
Pharmaceutical industry
Orphan device
Food and Drug Administration Amendments Act

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The Hard Truth About Rare Disease and Gene Therapy Drug Development

Added to Collection on: 2025-04-09 14:22:31

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Drug discovery
Pharmacy
Pharmaceutical industry
Pharmaceuticals policy
Biotechnology
Orphan drug
Biopharmaceutical
AstraZeneca
Rare disease
Medication
Clinical trial
Pfizer
Ken Getz

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