Tag: Orphan drug

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Recent Related Articles to Orphan drug

Global Orphan Drug Market Poised for Significant Growth Amid Rising Demand and ...

Added to Collection on: 2025-03-06 09:35:16

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Pharmaceuticals policy
Orphan drug
Pharmaceutical industry
Orphan Drug Act
Medication
Novartis
Biogen
Merck & Co.
Food and Drug Administration
Amgen
Specialty drugs in the United States
Prescription drug prices in the United States
United States

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Makary's 'Conditional Approval' Pathway for Rare Diseases Poses More Questions Than Answers

Added to Collection on: 2025-05-19 04:07:47

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Drug discovery
Food and Drug Administration
Clinical research
Design of experiments
Accelerated approval
Drug development
Ultragenyx
Aducanumab
Sarepta Therapeutics
Emil Kakkis
Marty Makary
Orphan drug
Vinay Prasad

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BioNTech SE: Orphan Drug Designation and Valuation Support Buy Rating - TipRanks.com

Added to Collection on: 2025-06-25 10:32:40

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Null
BioNTech
Pfizer-BioNTech COVID-19 vaccine
Orphan drug
Valuation
BioNTech SE
Robert Burns

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Biogen's Felzartamab in PMN: A Pioneering Play in Rare Kidney Disease Therapeutics

Added to Collection on: 2025-06-30 14:22:27

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Kidney diseases
Biogen
IgA nephropathy
Membranous glomerulonephritis
Accelerated approval
Kidney transplantation
Nephrology
Tacrolimus
Orphan drug
Nephrotic syndrome
Proteinuria
Kidney disease

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FDA Grants Orphan Status to UAB ALS Gene Therapy - Mirage News

Added to Collection on: 2025-07-15 11:21:03

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Rare diseases
Neurophysiology
Biogerontology
Klotho
ALS
Neuromuscular junction
Orphan drug
Klotho Neurosciences Inc.

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Regen BioPharma, Inc. to Seek Orphan Drug Designation for Lead Product - Yahoo Finance

Added to Collection on: 2025-07-23 12:15:30

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Stem cells
Hematopathology
Hematology
Oncology
Blood disorders
Aplastic anemia
Aplasia
Bone marrow
Anemia
Orphan drug
Hematopoietic stem cell transplantation
Chemotherapy
Regen BioPharma Inc.
HemaXellerate

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The High-Stakes Race for HAE: Ionis Pharmaceuticals and the Promise of Donidalorsen

Added to Collection on: 2025-08-21 17:49:49

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Spinal muscular atrophy
Antisense RNA
Huntington's disease
Ionis Pharmaceuticals
Hereditary angioedema
Ionis
Orphan drug
Medication

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Sanofi's Strategic Gambit in Rare Autoimmune Blood Disorders: Navigating High Barriers for ...

Added to Collection on: 2025-08-31 08:53:24

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Biotechnology
CAC 40
Sanofi
Pharmacy
AbbVie
Autoimmune disease
Gene therapy
Biopharmaceutical
Autoimmunity
Orphan drug

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Federal Legal Marijuana? MMJ's Billion Dollar Trajectory: We're Not Selling ... - Yahoo Finance

Added to Collection on: 2025-09-14 09:36:54

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Antiemetics
Appetite stimulants
Entheogens
Euphoriants
Aromatase inhibitors
Medical cannabis
Dronabinol
Cannabidiol
Tetrahydrocannabinol
Cannabinoid
Cannabis
Orphan drug

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FDA Grants Orphan Drug Designation to Biostar Pharma's Utidelone for Pancreatic Cancer ...

Added to Collection on: 2025-09-25 15:07:24

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Food and Drug Administration
Biotechnology law
Drug discovery
Orphan drug
Pharmaceuticals policy
Investigational New Drug
Pharmaceutical industry
Biostar Pharma Inc.
Mark Chiang
u.s. food and drug administration

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