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Radiology 182:437-444, Fritz,R.C.,et al, 1992
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JNNP 54:241-243, Liveson,J.A.,et al, 1991
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A 5-Year-Old Boy with Subacute and Refractory Myoclonus
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AAV9-Mediated Gene Therapy for Infantile-Onset Pompes Disease
NEJM 392:2438-2446, 2477, Ma,X.,et al, 2025
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NEJM 393:281-288, Hagerman,R.H. & Hagerman,P.J., 2025
Risdiplam in Presymptomatic Spinal Muscular Atrophy
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