BackgroundIn clinical research, the most appropriate way to assess the effect of an intervention is to conduct a randomized controlled trial (RCT). In the field of rare diseases, conducting an RCT is

Figshare2024-12-03 更新20

Safety and efficacy of Interferon y in Friedreich ataxia, using clinical and paraclinical indicators of disease

罕见病临床试验

神经退行性疾病药物

The data shared in the Zenodo repository include clinical and instrumental data from a randomized clinical trial open label phase II in Friedreich's Ataxia patients that underwent a 6 months treatment

Zenodo2019-05-22 更新10

Characteristics of completed and discontinued trials for rare diseases.

罕见病临床试验

研究特征分析

Characteristics of completed and discontinued trials for rare diseases.

Figshare2019-11-21 更新40

Additional file 4 of Profiling trial burden and patients’ attitudes to improve clinical research in epidermolysis bullosa

罕见病临床试验

患者经济负担

Additional file 4: Supplementary Fig. 4 Maximum extent of individual expenses considered acceptable for participation in a clinical trial. Graphical presentation of part four of the survey, asking for

Figshare2020-07-21 更新10

MPS III Participant Information.

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