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In two single-arm, open-label clinical trials comprising 37 children with MLD, Lenmeldy significantly reduced the risk of severe motor impairment or death compared with untreated children, according to the FDAs press release

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Side effects tend to peak during the first week at a new dose, then diminish

Most side effects improve substantially within 4-8 weeks of starting treatment or after each dose increase, and many patients find that the benefits of improved glycemic control and weight loss outweigh the temporary discomfort of initial side effects