RareConnect is a growing online network for rare disease communities, bringing together thousands of patients, families, and groups who might otherwise be isolated.
他们的研究有助于了解常见和罕见疾病的基础,并开发新的药物和治疗方法。
Their research helps to understand the underpinnings of common and rare diseases and illnesses, and to develop new drugs and treatments.
五种新药获得欧盟批准,其中包括两种针对罕见疾病的孤儿药。
Five therapies have been backed for approval in the European Union, including two orphan medicines addressing rare conditions.
作为临床研究协调员,她管理的多个观察,神经肌肉和神经退行性罕见疾病治疗和生物标志物研究。
As a clinical research coordinator, she managed multiple observational, therapeutic and biomarkers studies for neuromuscular and neurodegenerative rare disorders.
The partnership will see Cycle develop and commercialise 3D printed drugs for rare diseases, also known as‘orphan drugs.
也是因为基因组学的原因,Ashley和其他罕见疾病专家预期关于遗传基础方面疾病的文库将大幅扩大。
Because of genomics, Ashley and other rare disease specialists expect that the library of diseases with a genetic basis will drastically expand.
药物还接受孤儿药物指定,它提供奖励有助于和鼓励对罕见疾病药物的开发。
The drug also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.
由于基因组学,Ashley和其他罕见疾病的专家认为遗传疾病的“图书馆”将大幅扩大。
Because of genomics, Ashley and other rare disease specialists expect that the library of diseases with a genetic basis will drastically expand.
在天疱疮和类天疱疮(P/P)社区中,帮助这些罕见疾病的治疗并不总是理想的。
It's no secret among the pemphigus and pemphigoid(P/P) community that treatments to help these rare diseases are not always ideal.
具有侵袭性神经内分泌肿瘤(NETs)的患者治疗方案有限,很少有专门从事这种相对罕见疾病的肿瘤学家。
Patients with aggressive neuroendocrine tumours(NETs) have limited treatment options and there are few oncologists who are specialised in this relatively rare disease.
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