Abstract
We reflect on the worldwide research accomplishments, orphan product approvals, and the commitments by the rare diseases community. Major collaborative efforts by the public and private sectors increased interventions and diagnostics for rare diseases. We marvel at the impact of safe and effective treatments when they become available. Hope remains for the rare diseases community with a renewed commitment and acceptance of collaborative public-private partnerships of government translational research and regulatory programs, the biopharmaceutical, devices and diagnostics industries, academic investigators, and patient advocacy groups. The global rare diseases community remains strong and responsive to the voices and needs of patients, families, and clinicians awaiting diagnosis and treatment for their disease. We anticipate even greater successes in 2014 to reflect on Rare Disease Day 2015.
Highlights
We reflect on the worldwide research accomplishments, orphan product approvals, and the commitments by the rare diseases community
As the celebrations of Rare Disease Day occur throughout the world, we pause to reflect on the numerous and impressive research accomplishments and orphan product approvals granted during the past year
Not to be forgotten are the deep commitments required to reach these goals made by the rare diseases community including the pharmaceutical, biotechnology and medical devices and diagnostic industries, philanthropic foundations, patients and families, medical and scientific advisory boards associated with patient advocacy groups, the academic research community, research and regulatory scientists, government funding and regulatory agencies and the public
Summary
We reflect on the worldwide research accomplishments, orphan product approvals, and the commitments by the rare diseases community. Hope remains for the rare diseases community with a renewed commitment and acceptance of collaborative public-private partnerships of government translational research and regulatory programs, the biopharmaceutical, devices and diagnostics industries, academic investigators, and patient advocacy groups.
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