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1. 15.33 AVXS-101 in presymptomatic spinal muscular atrophy (SMA). Issue 12 (14th November 2019)

2. A Study of valproic acid for patients with spinal muscular atrophy. Issue 2 (25th November 2014)

5. Clinical spectrum of early onset epileptic encephalopathies caused by KCNQ2 mutation. Issue 7 (26th April 2013)

6. Clinical, biochemical and molecular analysis of 13 Japanese patients with β‐ureidopropionase deficiency demonstrates high prevalence of the c.977G > A (p.R326Q) mutation. Issue 5 (14th February 2014)

8. Erratum to: Clinical, biochemical and molecular analysis of 13 Japanese patients with β‐ureidopropionase deficiency demonstrates high prevalence of the c.977G > A (p.R326Q) mutation. Issue 6 (12th August 2014)