Search results: Sage Journals

Open accessReview articleFirst published November, 2024

Inherited retinal dystrophies and orphan designations in the European Union

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

European Journal of Ophthalmology

Restricted accessOtherFirst published January, 1998

Orphan Drug Development — International Program and Study Design Issues

Marlene E. Haffner

Therapeutic Innovation & Regulatory Science

Restricted accessResearch articleFirst published February, 2001

The EU challenges on the designation of orphan medicinal products

Josep Torrent-Farnell, Rosa Morros

Pharmaceuticals Policy and Law

Restricted accessOtherFirst published July, 2000

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

Makoto Shiragami, Kiyohito Nakai

Therapeutic Innovation & Regulatory Science

Restricted accessResearch articleFirst published February, 2010

Efforts to develop orphan drugs: The FDA experience

Linda C. Ulrich

Pharmaceuticals Policy and Law

Restricted accessOtherFirst published January, 1998

Designing Clinical Trials to Study Rare Disease Treatment

Marlene E. Haffner

Therapeutic Innovation & Regulatory Science

Restricted accessResearch articleFirst published February, 2010

Scientific advice for orphan drugs: Critical points and perspectives

Jordi Llinares Garcia

Pharmaceuticals Policy and Law

Restricted accessResearch articleFirst published February, 2010

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

Chris Walker

Pharmaceuticals Policy and Law

Restricted accessResearch articleFirst published February, 2010

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

Chris Walker

Pharmaceuticals Policy and Law

Open accessResearch articleFirst published March, 2023

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

Open accessReview articleFirst published November, 2024

Inherited retinal dystrophies and orphan designations in the European Union

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

European Journal of Ophthalmology

Restricted accessOtherFirst published January, 1998

Orphan Drug Development — International Program and Study Design Issues

Marlene E. Haffner

Therapeutic Innovation & Regulatory Science

Restricted accessResearch articleFirst published February, 2001

The EU challenges on the designation of orphan medicinal products

Josep Torrent-Farnell, Rosa Morros

Pharmaceuticals Policy and Law

Restricted accessOtherFirst published July, 2000

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

Makoto Shiragami, Kiyohito Nakai

Therapeutic Innovation & Regulatory Science

Restricted accessResearch articleFirst published February, 2010

Efforts to develop orphan drugs: The FDA experience

Linda C. Ulrich

Pharmaceuticals Policy and Law

Restricted accessOtherFirst published January, 1998

Designing Clinical Trials to Study Rare Disease Treatment

Marlene E. Haffner

Therapeutic Innovation & Regulatory Science

Restricted accessResearch articleFirst published February, 2010

Scientific advice for orphan drugs: Critical points and perspectives

Jordi Llinares Garcia

Pharmaceuticals Policy and Law

Restricted accessResearch articleFirst published February, 2010

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

Chris Walker

Pharmaceuticals Policy and Law

Restricted accessResearch articleFirst published February, 2010

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

Chris Walker

Pharmaceuticals Policy and Law

Open accessResearch articleFirst published March, 2023

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