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1-10 of 100 results for Orphan designation

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Select article: Inherited retinal dystrophies and orphan designations in the European Union

Open access

Review article

First published 2024-11

Inherited retinal dystrophies and orphan designations in the European Union

Jane Moseley, Tim Leest, Kristina Larsson [...]

European Journal of Ophthalmology

Select article: Orphan Drug Development — International Program and Study Design Issues

Retricted access

Other

First published 1998-1

Orphan Drug Development — International Program and Study Design Issues

Marlene E. Haffner

Therapeutic Innovation & Regulatory Science

Select article: The EU challenges on the designation of orphan medicinal products

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Research article

First published 2001-2

The EU challenges on the designation of orphan medicinal products

Josep Torrent-Farnell, Rosa Morros

Pharmaceuticals Policy and Law

Select article: Development of Orphan Drugs in Japan: Characteristics of Orphan Drugs Developed in Japan *

Retricted access

Other

First published 2000-7

Development of Orphan Drugs in Japan: Characteristics of Orphan Drugs Developed in Japan *

Makoto Shiragami, Kiyohito Nakai

Therapeutic Innovation & Regulatory Science

Select article: Efforts to develop orphan drugs: The FDA experience

Retricted access

Research article

First published 2010-2

Efforts to develop orphan drugs: The FDA experience

Linda C. Ulrich

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: Scientific advice for orphan drugs: Critical points and perspectives

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Research article

First published 2010-2

Scientific advice for orphan drugs: Critical points and perspectives

Jordi Llinares Garcia

Pharmaceuticals Policy and Law

Select article: Designation,plausibility,protocol assistance,clinical benefit,similarity,reassessment: Rules and experiences

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Research article

First published 2010-2

Designation,plausibility,protocol assistance,clinical benefit,similarity,reassessment: Rules and experiences

Chris Walker

Pharmaceuticals Policy and Law

Select article: ODs assessment in the centralized procedure: Conditional and exceptional approvals and follow up measures – Rules and experiences

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Research article

First published 2010-2

ODs assessment in the centralized procedure: Conditional and exceptional approvals and follow up measures – Rules and experiences

Chris Walker

Pharmaceuticals Policy and Law

Select article: Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience

Open access

Research article

First published 2023-03

Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience

Richa Madan Lomash, Oleg Shchelochkov, Randy J. Chandler [...]

Human Gene Therapy

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