Efficacy and safety of cyclic pyranopterin monophosphate substitution in severe molybdenum cofactor deficiency type A: a prospective cohort study

Bernd C Schwahn, Francjan J Van Spronsen, Abdel A Belaidi, Stephen Bowhay, John Christodoulou, Terry G Derks, Julia B Hennermann, Elisabeth Jameson, Kai Koenig, Tracy L McGregor, Esperanza Font-Montgomery, Jose A Santamaria-Araujo, Saikat Santra, Mamta Vaidya, Anne Vierzig, Evangeline Wassmer, Ilona Weis, Flora Y Wong, Alex Veldman, Guenter Schwarz