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13,14,15,16,17,18 Currently, the disease can only be diagnosed by DNA sequencing, and only a subset of individuals have detectable reductions in Asn levels in serum and cerebrospinal fluid, hindering the use of this test for initial screening

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What to realistically expect in 36 months Human clinical trials showing benefit typically ran for several months

Hanai T, Shiraki M, Ohnishi S, et al