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October 25, 2019
Moira Gunn, PhD, professor of bioentrepreneurship at the University of San Francisco, discusses the biggest hurdles those in healthcare face when looking to create new therapies for rare diseases.
Dawn Rotellini, COO of the National Hemophilia Foundation, discusses how recent policy changes seeking to improve access to and create new therapies for patients with rare diseases.
October 24, 2019
A review of novel drug approvals in 2018 found more than half of all novel approvals were for rare diseases and that 2018 also saw more novel drug approvals for rare disease than any other year.
Dawn Rotellini, chief operating officer of the National Hemophilia Foundation, discusses the important of including patient advocacy groups in annual meetings such as the NORD Summit.
A new analysis of an open-label extension study of the phase 3 PREVENT study is providing further evidence of the safety and efficacy of the treatment of the neuromyelitis optica spectrum disorder.
A new study presented at NORD Summit 2019 found rare disease is underrepresented in published literature and, more often than not, articles are not available in open-access journals.