Orphanet J Rare Dis
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Orphanet J Rare Dis · Feb 2014
A past with uncertainty, a future with hope--rare disease day 2014 from a USA perspective.
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. ⋯ 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.