Rare Community Profiles: PKD Foundation and IQVIA Partner to Create Next-Gen ADPKD Registry
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Rare Community Profiles: PKD Foundation and IQVIA Partner to Create Next-Gen ADPKD Registry

Rare Community Profiles   Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families,…

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FDA Grants Orphan Drug Designation to Ganaxolone for Lennox-Gastaut Syndrome
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FDA Grants Orphan Drug Designation to Ganaxolone for Lennox-Gastaut Syndrome

  Prior to the end of March 2023, ganaxolone had received six separate Orphan Drug designations from the FDA; more recently, as shared in a news release from commercial-stage pharmaceutical…

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