National Organization for Rare Diseases and Orphan Products Breakthrough Summit

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.

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.

Moira Gunn, PhD, professor of bioentrepreneurship at the University of San Francisco, discusses why it is important for primary care providers to keep up-to-date on rare disease developments.

Dawn Rotellini, chief operating officer of the National Hemophilia Foundation, discusses the impact recent advances in technology and changes in policy focusing on rare diseases have impacted the disease state of hemophilia.

Lucas Kempf, MD, associate director of the FDA's Rare Diseases Program, discusses the impact of recent policy changes that center around increasing access to and creating new therapies for rare disease.