National Organization for Rare Diseases and Orphan Products Breakthrough Summit

Latest from NORD's Voice of the Community

Moira Gunn, PhD: Challenges of Developing Rare Disease Therapies

October 25, 2019

Article

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: Impact of Policy Changes on Patients

October 25, 2019

Article

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.

Approvals for Rare Diseases Reached Historic High in 2018

October 24, 2019

Article

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: Importance of Patient Advocacy at Annual Meetings

October 24, 2019

Article

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.

Eculizumab for Neuromyelitis Optica Spectrum Disorder

October 24, 2019

Article

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.

Study Finds Rare Diseases Still Underrepresented in Medical Journals

October 24, 2019

Article

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.

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