Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe diseaseMarc Nicolino, Priya S. Kishnani, Hanna Mandel et al.|Genetics in Medicine|2009Cited by 292
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrateGeorge A. Díaz, Brendan Lee, Lauren Krivitzky et al.|Hepatology|2012Cited by 95