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Insights from Families and Caregivers Navigating Rare Diseases

To help drive research in the areas that matter most, investigators from the RDCRN asked families and caregivers to share their experiences.

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Statistical Solutions Improve Accuracy of Clinical Trials with Rescue Therapy in Myasthenia Gravis

July 7, 2025

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Graph showing data simulation to illustrate the impact of rescue therapy on trial results.

Pre-Doc Preparatory Program (P³) in Translational Research

June 23, 2025

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MGNet Announces 2025 Scholars

December 9, 2024

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Headshots of Dr. Sikorski and Dr. Wildberger

Former LDN Fellow Paves the Way for Earlier Diagnosis in Metachromatic Leukodystrophy

September 24, 2024

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FCDGC Announces 2023 Pilot & Feasibility Project Awardee and Career Enhancement Fellow

April 11, 2023

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A photo of Aryal Rajindra and Idris Wazeerud-din.

Training Program for Clinical Research in Rare Disease Launches New Session

November 9, 2022

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Screenshot from a presentation on the Rare Diseases Research R25 Curriculum Grant

CPIC Announces 2022 Pilot Project Award Recipient

October 26, 2022

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Early Stage Investigator Spotlight: Stephanie Sacharow Helps Patients with Phenylketonuria Overcome Barriers to Treatment

October 25, 2022

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