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• W4387310286 abstract "Facioscapulohumeral muscular dystrophy (FSHD) is a genetic disorder characterized by progressive muscle weakness leading to permanent disability. There are no curative treatments, however, there are several upcoming clinical trials testing new therapies in FSHD. This study aimed to explore the patient preferences of people with FSHD to ensure that clinical trials can be designed to include outcome measures that are relevant and important to patients. 1147 participants responded to an online survey, representing 26 countries across Europe and a range of disease severities. The study highlighted the key symptoms causing concern for FSHD patients - muscle weakness and mobility issues - reflecting what participants want targeted for future therapies. The need for clear information and communication throughout clinical trials was emphasised. Factors most encouraging trial participation included access to new investigational therapies, access to trial results and benefits for the FSHD community. Factors most discouraging trial participation included travel related issues and fear of side effects. The results from this study identified the unmet healthcare needs of FSHD patients and should provide researchers and industry with areas of therapeutic research that would be meaningful to patients, as well as supporting the development of patient centric outcome measures in clinical trials. Facioscapulohumeral muscular dystrophy (FSHD) is a genetic disorder characterized by progressive muscle weakness leading to permanent disability. There are no curative treatments, however, there are several upcoming clinical trials testing new therapies in FSHD. This study aimed to explore the patient preferences of people with FSHD to ensure that clinical trials can be designed to include outcome measures that are relevant and important to patients. 1147 participants responded to an online survey, representing 26 countries across Europe and a range of disease severities. The study highlighted the key symptoms causing concern for FSHD patients - muscle weakness and mobility issues - reflecting what participants want targeted for future therapies. The need for clear information and communication throughout clinical trials was emphasised. Factors most encouraging trial participation included access to new investigational therapies, access to trial results and benefits for the FSHD community. Factors most discouraging trial participation included travel related issues and fear of side effects. The results from this study identified the unmet healthcare needs of FSHD patients and should provide researchers and industry with areas of therapeutic research that would be meaningful to patients, as well as supporting the development of patient centric outcome measures in clinical trials." @default.
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• W4387310286 date "2023-10-01" @default.
• W4387310286 modified "2023-10-07" @default.
• W4387310286 title "P311 Facioscapulohumeral muscular dystrophy European patient survey: assessing patient preferences in clinical trial participation " @default.
• W4387310286 doi "https://doi.org/10.1016/j.nmd.2023.07.221" @default.
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