Groft Stephen C
Office of Rare Diseases Research, National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, MD 20892-4874, USA.
Orphanet J Rare Dis. 2014 Feb 28;9:31. doi: 10.1186/1750-1172-9-31.
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.
我们回顾了全球范围内的研究成果、罕见病产品批准情况以及罕见病群体所做出的努力。公共和私营部门的重大合作努力增加了对罕见病的干预措施和诊断方法。当安全有效的治疗方法问世时，我们惊叹于它们所产生的影响。对于罕见病群体来说，随着政府转化研究和监管项目、生物制药、器械和诊断行业、学术研究人员以及患者倡导组织重新承诺并接受公私合作关系，希望犹存。全球罕见病群体依然强大，并能响应患者、患者家庭以及等待疾病诊断和治疗的临床医生的声音和需求。我们期待在2014年取得更大的成功，以便在2015年罕见病日进行回顾。
