August 5, 2019
Rare Reading Before You Head to the 2019 RARE Patient Advocacy Summit
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August 2, 2019
Ultragenyx Seeks FDA Approval for Long-Chain Fatty Acid Oxidation Disorders Therapy
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July 29, 2019
FDA Grants Orchard RMAT Designation for Wiskott-Aldrich Syndrome Gene Therapy
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July 1, 2019
Poll Finds Strong Support for Gene Therapies Despite High Cost
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July 1, 2019
Zogenix Reaches Agreement with FDA for Resubmission of Dravet Drug Application
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June 25, 2019
Buoyed by Positive Gene Therapy Results in DEB, Krystal Raises $100 Million
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June 13, 2019
Rare Leader: Jeff Woytovich, Founder, Children’s Alopecia Project
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June 12, 2019
Sobi Acquires Full Rights to Approved HLH Drug from Novimmune
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June 6, 2019
Rare Leader: Sandra Bedrosian-Sermone, ADNP Kids Research Foundation
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May 23, 2019
Rare Leader: Rob Long, Executive Director, Uplifting Athletes
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May 17, 2019
Understanding the Role of Natural History Studies in Clinical Trials
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