Rare Diseases
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Medical Nutrition Equity Act Capitol Hill Day Planned for June 1
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More Than 100 Patient Organizations Join NORD in Supporting the RARE Act
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RFPs Available for the Study of Three Rare Diseases
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2018 Marks 35th Anniversary of NORD and the Orphan Drug Act
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Register Now for NORD’s Rare Impact Awards Celebration
From the Journals
Study supports meningococcal B vaccine in children with rare diseases
Its strength held up in children with asplenia and splenic dysfunction but flagged in complement deficiency
Latest News
Oral drug seen preventing angioedema attacks
A novel agent led to a nearly 75% reduction in monthly attacks among people with a rare genetic disorder.
News
FDA approves Epidiolex for Lennox-Gastaut syndrome and Dravet syndrome
The approval, the first for a marijuana-derived pharmaceutical product, falls in line with the unanimous recommendation of an FDA advisory panel...
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FDA approves rituximab for treating pemphigus vulgaris
Approval was based on a prospective, multicenter, open-label, randomized study conducted in France.