Last Updated: 09/26/2026
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Found 8 publications
From roadmap to a sustainable end-to-end individualized therapy pathway.
Journal: Therapeutic advances in rare disease
Published: November 28, 2024
The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap.
Journal: Nature reviews. Drug discovery
Published: September 19, 2024
FDA/Arthritis Foundation osteoarthritis drug development workshop recap: Assessment of long-term benefit.
Journal: Seminars in arthritis and rheumatism
Published: June 08, 2022
Prenatal Somatic Cell Gene Therapies: Charting a Path Toward Clinical Applications (Proceedings of the CERSI-FDA Meeting).
Journal: Journal of clinical pharmacology
Published: May 20, 2022
Clinical Development of Gene Therapies: The First Three Decades and Counting.
Journal: Molecular therapy. Methods & clinical development
Published: November 19, 2020
Gene and Stem Cell Therapies for Fetal Care: A Review.
Journal: JAMA pediatrics
Published: June 30, 2020
Regenerative Medicine Therapies for Rare Diseases.
Journal: Translational science of rare diseases
Published: January 08, 2019
Investigational New Drug applications: a 1-year pilot study on rates and reasons for clinical hold.
Journal: Journal of investigative medicine : the official publication of the American Federation for Clinical Research
Published: February 26, 2016
Showing 1-8 of 8
Last Updated: 09/26/2026