Ways to Engage

Join the Portal

Take the next step to connect with the rare disease community. Create a profile to connect with others, read resources and post to the event hub.

Share Your Story

It’s our experiences that shapes you and inspires others. Share your rare story with us.

Events

RARE Patient Advocacy Summit
From: 09/21/2020 To: 09/23/2020
08:00 AM - 05:00 PM UTC-12:00
1380 Harbor Island Drive, San Diego, California, United States of America
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Resources

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News

January 21, 2020
Stealth Initiates Clinical Study of Second-Generation Mitochondrial Candidate in Neurodegenerative Diseases
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January 20, 2020
FDA to Move to Online Submissions for Orphan Drug Designation Requests
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January 17, 2020
Driving N-of-1 Therapies for Ultra-Rare Disease Patients
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January 17, 2020
Creating the Means to an End
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January 16, 2020
Rare Leader: Daniel Lewi, Founder and CEO, Cure & Action for Tay-Sachs Foundation
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January 16, 2020
FDA Clears GeneTx and Ultragenyx to Begin Angelman Syndrome Trial
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