Latest News

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

A new pilot study funded by the Myasthenia Gravis Rare Disease Network (MGNet) explores how statistical models can help improve clinical trials.

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

Congenital Disorders of Glycosylation Over Time: What We’re Learning from a Natural History Study

March 22, 2023

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Congenital Disorders of Glycosylation Over Time: What We’re Learning from a Natural History Study

Patient Spotlight: Franklin and List Families Navigate Life with Congenital Disorder of Glycosylation

June 30, 2022

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The Franklin family at a playground, and Dee List wearing a graduation cap

Patient Spotlight: Estopinan Family Advocates for TK2-Related Mitochondrial DNA Depletion Syndrome

May 26, 2022

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The Estopinan family at an outdoor gathering

Patient Spotlight: Billy Anton Balances Management and Lifestyle with Primary Ciliary Dyskinesia

March 29, 2022

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Patient Spotlight: Felipe Polo-Wood Forges Ahead with Myasthenia Gravis

February 25, 2022

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Patient Spotlight: Mitchell Magyar Makes an Impact on Urea Cycle Disorder Research

December 16, 2021

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Mitchell Magyar with his mother and sister, wearing sunglasses and smiling

Patient Spotlight: Fynan Family Fueled by OI Research

November 16, 2021

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Michelle Fynan, PhD, LMHC

Patient Spotlight: Anthony Anzell Fights Hereditary Hemorrhagic Telangiectasia with Endurance and Education

October 19, 2021

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Anthony Anzell, PhD