February 15, 2019
Passage Bio Raises $115.5 Million to Develop Gene Therapies for CNS Disease
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February 15, 2019
Rare Leader: Lori Sames, CEO, Hannah’s Hope Fund
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February 7, 2019
Rare Leader: Liz Marfia-Ash, President and Founder, GRIN2B Foundation
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February 5, 2019
Sen. Bernie Sanders Investigates $375,000 Price of LEMS Drug
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January 10, 2019
NEJM Study Shows Positive Results of Sanofi’s Drug for Rare Blood Disorder
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December 21, 2018
FDA Approves Stemline Therapeutics BPDCN Therapy Elzonris
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December 20, 2018
Rare Leader: Brad Crittenden, Executive Director, Canadian Association of Pompe
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December 17, 2018
Undiagnosed Rare Disease Patients Cost NHS England $4.3 Billion, and then Some
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October 30, 2018
Study Shows Cholic Acid Replacement Leads to Favorable Outcome in Adulthood for Children&hellip...
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October 26, 2018
Conquering Life with a Rare Disease
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September 26, 2018
CRO Synteract Expands Team with Rare Disease Experts
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September 25, 2018
Ferris Bueller and the Cost of Pivotal Clinical Trials
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