Rare Diseases
Article
Medical Nutrition Equity Act Capitol Hill Day Planned for June 1
Article
More Than 100 Patient Organizations Join NORD in Supporting the RARE Act
Article
RFPs Available for the Study of Three Rare Diseases
Article
2018 Marks 35th Anniversary of NORD and the Orphan Drug Act
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Register Now for NORD’s Rare Impact Awards Celebration
Conference Coverage
Study offers snapshot of esophageal strictures in EB patients
LAKE TAHOE, CALIF. – An estimated 10%-17% of EB patients experience strictures, with an overrepresentation in the recessive dystrophic EB subtype...
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
Conference Coverage
Limited anakinra course often enough for systemic JIA if used first
AMSTERDAM – About two-thirds of children with JIA remained in remission after 5 years when a 3-month course of the IL-1 receptor antagonist...
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...
Video
Maintenance chemo boosts survival for youth with high-risk rhabdomyosarcoma
CHICAGO - Lead study author Dr. Gianni Bisogno discusses the practice changing results and describes plans for a new trial to explore alternate...
Conference Coverage
Myelin antibody predicts ADEM relapse
LOS ANGELES – Could results help to guide decisions on immunosuppressive treatment?