Karl E. Mengel
Hochheim, TH, Germany
Last Updated: 09/26/2026
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Found 18 publications
Long-term efficacy and safety of arimoclomol in Niemann-Pick disease type C: Final results of the phase 2/3 NPC-002 48-month open-label extension trial.
Journal: Molecular genetics and metabolism
Published: March 17, 2025
One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency.
Journal: Genetics in medicine : official journal of the American College of Medical Genetics
Published: October 07, 2022
Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease.
Journal: Neurology
Published: August 23, 2021
Correction to: Clinical disease progression and biomarkers in Niemann-Pick disease type C: a prospective cohort study.
Journal: Orphanet journal of rare diseases
Published: June 02, 2021
Brine residues and organics in the Urvara basin on Ceres.
Journal: Nature communications
Published: May 17, 2021
One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency.
Journal: Genetics in medicine : official journal of the American College of Medical Genetics
Published: January 19, 2021
Prospective study of the natural history of chronic acid sphingomyelinase deficiency in children and adults: eleven years of observation.
Journal: Orphanet journal of rare diseases
Published: December 01, 2020
Clinical disease progression and biomarkers in Niemann-Pick disease type C: a prospective cohort study.
Journal: Orphanet journal of rare diseases
Published: June 11, 2020
Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study.
Journal: Neuromuscular disorders : NMD
Published: November 22, 2017
Corrigendum to "Cause of death in patients with chronic visceral and chronic neurovisceral acid sphingomyelinase deficiency (Niemann-Pick disease type B and B variant): Literature review and report of new cases" [Mol. Genet. Metab. 118 (2016) 206-213].
Journal: Molecular genetics and metabolism
Published: November 14, 2017
Long-term outcomes of systemic therapies for Hurler syndrome: an international multicenter comparison.
Journal: Genetics in medicine : official journal of the American College of Medical Genetics
Published: October 19, 2017
Last Updated: 09/26/2026