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Articles (188)

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1-10 of 188 results for Orphan disease

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Select article: From care to cure: a patient engagement framework for rare disease and orphan drug research

Open access

Research article

First published 2025

From care to cure: a patient engagement framework for rare disease and orphan drug research

Nahya Awada, Anil Varughese

Therapeutic Advances in Rare Disease

Select article: Interstitial Lung Disease in Children: An Orphan Lung Disease

Retricted access

Research article

First published 1994-6

Interstitial Lung Disease in Children: An Orphan Lung Disease

Bettina C. Hilman

Pediatric Allergy,Immunology,and Pulmonology

Select article: Tyzzer disease in 19 preweaned orphaned kittens

Open access

Brief report

First published 2023-3

Tyzzer disease in 19 preweaned orphaned kittens

Sai Fingerhood, Fábio S. Mendonça, Francisco A. Uzal [...]

Journal of Veterinary Diagnostic Investigation

Select article: The Orphan Drug Act: An Appropriate Approval Pathway for Treatments of Rare Diseases?

Retricted access

Research article

First published 2019-10

The Orphan Drug Act: An Appropriate Approval Pathway for Treatments of Rare Diseases?

Michael Gabay

Hospital Pharmacy

Select article: Orphan Drugs: The Current Situation in the United States,Europe,and Asia

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Research article

First published 1997-1

Orphan Drugs: The Current Situation in the United States,Europe,and Asia

Abbey S. Meyers

Therapeutic Innovation & Regulatory Science

Select article: Hidradenitis Suppurativa: A Novel Model of Care and an Integrative Strategy to Adopt an Orphan Disease

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Review article

First published 2018-1

Hidradenitis Suppurativa: A Novel Model of Care and an Integrative Strategy to Adopt an Orphan Disease

Wayne Gulliver, Ian D. R. Landells, David Morgan [...]

Journal of Cutaneous Medicine and Surgery

Select article: Orphan drugs in haematology

Retricted access

Research article

First published 2010-2

Orphan drugs in haematology

Paola Zanon

Pharmaceuticals Policy and Law

Select article: Designing Clinical Trials to Study Rare Disease Treatment

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Other

First published 1998-1

Designing Clinical Trials to Study Rare Disease Treatment

Marlene E. Haffner

Therapeutic Innovation & Regulatory Science

Select article: Orphan Drugs: The United States Experience

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Other

First published 1999-4

Orphan Drugs: The United States Experience

Marlene E. Haffner

Therapeutic Innovation & Regulatory Science

Select article: The United States Orphan Drug ACT: Challenges and Success

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Research article

First published 1997-1

The United States Orphan Drug ACT: Challenges and Success

Marlene E. Haffner

Therapeutic Innovation & Regulatory Science

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