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

Comprehensive long-term efficacy and safety of recombinant human alpha-mannosidase (velmanase alfa) treatment in patients with alpha-mannosidosis

(2018) Allan M. Lund et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Alpha-mannosidosis: correlation between phenotype, genotype and mutant MAN2B1 subcellular localisation

(2015) Line Borgwardt et al.   Orphanet Journal of Rare Diseases

Systematic review of timed stair tests

(2014) Elizabeth Jean Nightingale et al.   JOURNAL OF REHABILITATION RESEARCH AND DEVELOPMENT

Enzyme replacement therapy for alpha-mannosidosis: 12 months follow-up of a single centre, randomised, multiple dose study

(2013) L. Borgwardt et al.   JOURNAL OF INHERITED METABOLIC DISEASE

The natural course and complications of alpha-mannosidosis—a retrospective and descriptive study

(2013) Dag Malm et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Effects of idursulfase enzyme replacement therapy for Mucopolysaccharidosis type II when started in early infancy: Comparison in two siblings

(2013) Go Tajima et al.   MOLECULAR GENETICS AND METABOLISM

Early initiation of enzyme replacement therapy for the mucopolysaccharidoses

(2013) Joseph Muenzer   MOLECULAR GENETICS AND METABOLISM

Natural history of alpha mannosidosis a longitudinal study

(2013) Michael Beck et al.   Orphanet Journal of Rare Diseases

Bronchial Responsiveness in Patients with Restrictive Spirometry

(2013) Jean I. Keddissi et al.   Biomed Research International

Enzyme replacement therapy for mucopolysaccharidosis II from 3 months of age: a 3-year follow-up

(2011) A Tylki- Szymanska et al.   ACTA PAEDIATRICA

Allogeneic hematopoietic SCT for alpha-mannosidosis: an analysis of 17 patients

(2011) M Mynarek et al.   BONE MARROW TRANSPLANTATION

Enzyme-Replacement Therapy in a 5-Month-Old Boy With Attenuated Presymptomatic MPS I: 5-Year Follow-up

(2009) O. Gabrielli et al.   PEDIATRICS