Last Updated: 09/26/2026
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Found 16 publications
Recommendations for the use of clinical outcome assessments in rare disease drug development.
Journal: EClinicalMedicine
Published: March 05, 2026
Unlocking the potential of disease prevention through regulatory science.
Journal: Nature reviews. Drug discovery
Published: February 10, 2026
The Rare Therapies Launchpad: a pilot program for individualized medicines in the UK.
Journal: Nature medicine
Published: March 11, 2025
Personalized medicine: when the common becomes the rare.
Journal: Frontiers in medicine
Published: November 06, 2024
Drug-device combinations in rare diseases: Challenges and opportunities.
Journal: Drug discovery today
Published: October 02, 2024
IRDiRC Drug Repurposing Guidebook: making better use of existing drugs to tackle rare diseases.
Journal: Nature reviews. Drug discovery
Published: October 23, 2023
Defining rare conditions in the era of personalized medicine.
Journal: Nature reviews. Drug discovery
Published: September 08, 2023
Harnessing the patient voice in real-world evidence: the essential role of patient-reported outcomes.
Journal: Nature reviews. Drug discovery
Published: October 02, 2019
Eligibility criteria in haematological cancer clinical trials-what's needed?
Journal: The Lancet. Haematology
Published: June 04, 2019
Last Updated: 09/26/2026