Christine í Dali

Efficacy and safety of arimoclomol in <scp>Niemann‐Pick</scp> disease type C: Results from a double‐blind, randomised, placebo‐controlled, multinational phase 2/3 trial of a novel treatment

Eugen Mengel, Marc C. Patterson, Rosalia Maria Da Riol et al. · Journal of Inherited Metabolic Disease · 2021 · 99 citations · Full text

Life Sciences, Pharmacology, Pharmacotherapy +17

Metachromatic leukodystrophy: natural course of cerebral MRI changes in relation to clinical course

Samuel Groeschel, Christiane Kehrer, Corinna Engel et al. · Journal of Inherited Metabolic Disease · 2011 · 93 citations · Full text

Medicine, Neurology, Radiology +11

Annual severity increment score as a tool for stratifying patients with Niemann-Pick disease type C and for recruitment to clinical trials

Mario Cortina‐Borja, Daniëlle te Vruchte, Eugen Mengel et al. · Orphanet Journal of Rare Diseases · 2018 · 81 citations · Full text

Medicine, Clinical Sciences, Neurology +14

Safety of intrathecal delivery of recombinant human arylsulfatase A in children with metachromatic leukodystrophy: Results from a phase 1/2 clinical trial

Christine í Dali, Caroline Sevin, Ingeborg Krägeloh‐Mann et al. · Molecular Genetics and Metabolism · 2020 · 72 citations · Full text

Medicine, Neurology, Pediatrics +11

Efficacy and safety of Velmanase alfa in the treatment of patients with alpha‐mannosidosis: results from the core and extension phase analysis of a phase III multicentre, double‐blind, randomised, placebo‐controlled trial

Line Borgwardt, Nathalie Guffon, Yasmina Amraoui et al. · Journal of Inherited Metabolic Disease · 2018 · 62 citations · Full text

Pharmacy, Pharmacology, Pharmacotherapy +7