Last Updated: 09/26/2026
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Found 7 publications
Correction to: Analysis of the first ten years of FDA's rare pediatric disease priority review voucher program: designations, diseases, and drug development.
Journal: Orphanet journal of rare diseases
Published: March 22, 2024
Trends in drug development for amyotrophic lateral sclerosis.
Journal: Nature reviews. Drug discovery
Published: December 12, 2023
Analysis of the first ten years of FDA's rare pediatric disease priority review voucher program: designations, diseases, and drug development.
Journal: Orphanet journal of rare diseases
Published: December 08, 2023
Determining Commonalities in the Experiences of Patients with Rare Diseases: A Qualitative Analysis of US Food and Drug Administration Patient Engagement Sessions.
Journal: The patient
Published: September 26, 2023
A comprehensive study of the rare diseases and conditions targeted by orphan drug designations and approvals over the forty years of the Orphan Drug Act.
Journal: Orphanet journal of rare diseases
Published: April 14, 2023
Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases.
Journal: Orphanet journal of rare diseases
Published: October 18, 2020
Common Filing Deficiencies in Abbreviated New Drug Applications Containing Clinical Endpoint Studies.
Journal: Therapeutic innovation & regulatory science
Published: May 04, 2018
Showing 1-7 of 7
Last Updated: 09/26/2026