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Dystonia Coalition: Advice and Accomplishments from 15 Years of Dystonia Research

Principal Investigator Hyder Jinnah, MD, PhD, shares advice and accomplishments from 15 years of dystonia research.

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Developing Best Practices for Data Sharing in Rare Disease Research

September 26, 2025

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Chart showing agreements among the RDCRN parties involved in data sharing.

Insights from Families and Caregivers Navigating Rare Diseases

August 27, 2025

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$27M Grant Renews Cincinnati Children’s as Coordinating Center for the Rare Diseases Clinical Research Network

August 12, 2025

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Group photo of approximately 50 DMCC members seated and standing in rows.

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.

Porphyrias Over Time: What We’re Learning from a Natural History Study

December 17, 2024

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Porphyrias Over Time: What We're Learning from a Natural History Study

Leukodystrophy Over Time: What We’re Learning from a Natural History Study

November 26, 2024

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Leukodystrophy Over Time: What We’re Learning from a Natural History Study

Artificial Intelligence-Powered Telemedicine Tool Automates Scoring of Neuromuscular Examinations in Myasthenia Gravis

October 28, 2024

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Screenshot of the graphic user interface used by doctors for remote examinations. Source: Bioengineering journal

Pompe Disease Over Time: What We’re Learning from a Natural History Study

September 25, 2024

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Pompe Disease Over Time: What We're Learning from a Natural History Study