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https://doi.org/10.1016/j.jval.2023.03.586
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Cite Journal: Value in Health |  Publication Date: Jun 1, 2023 |
MLD is an ultra-rare, rapidly progressive neurodegenerative disease, which leads to motor and cognitive decline, resulting in increasingly poor quality of life and premature death. This study evaluated the face validity of MLD specific utility values in relation to published utility values for other severe rare genetic diseases.
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