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Failing to slow disease Progression of disease was measured by the change in Clinical Dementia Rating Sum of Boxes (CDR-SB) score and compared to placebo on top of standard of care
23 A phase 1 trial of NTLA-2001 demonstrated dose-dependent reductions in serum TTR, with reductions of 52% and 87% at low and high doses, respectively
In most cases, it is not understood whether these diseases are caused by the extra chromosome, the altered metabolic profile, or both
Early recognition and treatment of these deficiencies can help prevent and manage various neuropsychiatric conditions, thereby enhancing overall mental health (6)
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