Christine I. Dali
Copenhagen, Denmark
Last Updated: 09/26/2026
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Found 23 publications
Long-term real-world safety and effectiveness of arimoclomol in individuals with NPC: Outcomes from the US early access program over a 4-year period.
Journal: Molecular genetics and metabolism
Published: April 13, 2026
Arimoclomol in infants with Niemann-Pick disease type C: Results from the phase 2/3 open-label pediatric substudy.
Journal: Molecular genetics and metabolism reports
Published: November 20, 2025
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
Efficacy results from a 12-month double-blind randomized trial of arimoclomol for treatment of Niemann-Pick disease type C (NPC): Presenting a rescored 4-domain NPC Clinical Severity Scale.
Journal: Molecular genetics and metabolism reports
Published: February 10, 2025
Effects of sulfatide on peripheral nerves in metachromatic leukodystrophy.
Journal: Annals of clinical and translational neurology
Published: August 25, 2023
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
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.
Journal: Journal of inherited metabolic disease
Published: June 16, 2017
Comprehensive long-term efficacy and safety of recombinant human alpha-mannosidase (velmanase alfa) treatment in patients with alpha-mannosidosis.
Journal: Journal of inherited metabolic disease
Published: June 16, 2017
The ethical framework for performing research with rare inherited neurometabolic disease patients.
Journal: European journal of pediatrics
Published: September 09, 2016
A Novel TTBK2 De Novo Mutation in a Danish Family with Early-Onset Spinocerebellar Ataxia.
Journal: Cerebellum (London, England)
Published: May 12, 2016
Erratum to: Review and evaluation of the methodological quality of the existing guidelines and recommendations for inherited neurometabolic disorders.
Journal: Orphanet journal of rare diseases
Published: April 18, 2016
Metachromatic Leukodystrophy: An Assessment of Disease Burden.
Journal: Journal of child neurology
Published: January 19, 2016
Last Updated: 09/26/2026