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Patient families expressed the following: Long-term goal is independenceFunctional independence and personal autonomy are key outcomes with the greatest impact for improving quality of life Incremental therapiesFirst slow, then halt, disease progression, then work to reverse symptoms Sustainable therapiesLifelong diseases need lifelong treatments, therefore the therapies we identify must be available to patients for as many years as they need, and have a minimized financial burden II

10.1016/j.semcdb.2017.5.023 94 MoloneyJ

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10.1016/S0022-510X(97)00110-X 191 Rodrguez-CarballoE.GmezB.VenturaF

Research has shown that many alkaloids have qualities that alleviate pain