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You searched for: Author/Creator Shieh, Perry B.

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1. Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study. Issue 2 (21st January 2016)

2. Congenital myasthenic syndrome caused by a frameshift insertion mutation in GFPT1. (August 2020)

3. Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post HOC analysis from the ACT DMD trial. Issue 5 (27th September 2018)

4. Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta‐analysis of disease progression rates in recent multicenter clinical trials. Issue 1 (7th November 2019)

7. Editorial by concerned physicians: Unintended effect of the orphan drug act on the potential cost of 3, 4‐diaminopyridine. Issue 2 (21st December 2015)

10. Natural history of infantile‐onset spinal muscular atrophy. Issue 6 (8th December 2017)