Vĕra Malinová

Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial

Jordi Díaz‐Manera, Priya S. Kishnani, Hani Kushlaf et al. · The Lancet Neurology · 2021 · 169 citations · Full text

Late-onset Pompe Disease, Autoimmune Disease, Clinical Infectious Disease +3

Clinical Effect And Safety Profile of Recombinant Human Lysosomal Acid Lipase in Patients With Cholesteryl Ester Storage Disease

Manisha Balwani, Catherine Breen, Gregory M. Enns et al. · Hepatology · 2013 · 116 citations · Full text

Clinical Features of Lysosomal Acid Lipase Deficiency

Barbara K. Burton, Patrick Deegan, Gregory M. Enns et al. · Journal of Pediatric Gastroenterology and Nutrition · 2015 · 111 citations · Full text

Acetyl- <scp>dl</scp> -leucine in Niemann-Pick type C

Tatiana Brémovà-Ertl, Vĕra Malinová, Yasmina Amraoui et al. · Neurology · 2015 · 100 citations

Sebelipase alfa over 52weeks reduces serum transaminases, liver volume and improves serum lipids in patients with lysosomal acid lipase deficiency

Vassili Valayannopoulos, Vĕra Malinová, Tomáš Honzík et al. · Journal of Hepatology · 2014 · 95 citations · Full text