Nonclinical comparability studies of recombinant human arylsulfatase A addressing manufacturing process changes

Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial

(2016) Maria Sessa et al.   LANCET

Intrathecal delivery of recombinant human arylsulfatase A in children with late-infantile metachromatic leukodystrophy

(2016) Christine Dali et al.   MOLECULAR GENETICS AND METABOLISM

Long-term outcomes after allogeneic hematopoietic stem cell transplantation for metachromatic leukodystrophy: the largest single-institution cohort report

(2015) Alexander A. Boucher et al.   Orphanet Journal of Rare Diseases

Pressure for drug development in lysosomal storage disorders – a quantitative analysis thirty years beyond the US orphan drug act

(2015) Konstantin Mechler et al.   Orphanet Journal of Rare Diseases

Intrathecal delivery of protein therapeutics to the brain: A critical reassessment

(2014) Pericles Calias et al.   PHARMACOLOGY & THERAPEUTICS

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

(2013) A. Biffi et al.   SCIENCE

Correction of Brain Oligodendrocytes by AAVrh.10 Intracerebral Gene Therapy in Metachromatic Leukodystrophy Mice

(2012) Françoise Piguet et al.   HUMAN GENE THERAPY

CNS Penetration of Intrathecal-Lumbar Idursulfase in the Monkey, Dog and Mouse: Implications for Neurological Outcomes of Lysosomal Storage Disorder

(2012) Pericles Calias et al.   PLoS One

Developing treatment options for metachromatic leukodystrophy

(2011) Spyros P. Batzios et al.   MOLECULAR GENETICS AND METABOLISM

Safety Evaluation of Chronic Intrathecal Administration of Idursulfase-IT in Cynomolgus Monkeys

(2011) Brian R. Felice et al.   TOXICOLOGIC PATHOLOGY

Safety evaluation of chronic intrathecal administration of heparan N-sulfatase in juvenile cynomolgus monkeys

(2011) Richard W. Pfeifer et al.   Drug Delivery and Translational Research

Metachromatic Leukodystrophy – An Update

(2010) V. Gieselmann et al.   NEUROPEDIATRICS

Metachromatic Leukodystrophy: A Case of Triplets With the Late Infantile Variant and a Systematic Review of the Literature

(2009) Asif Mahmood et al.   JOURNAL OF CHILD NEUROLOGY

Enzyme Replacement Improves Ataxic Gait and Central Nervous System Histopathology in a Mouse Model of Metachromatic Leukodystrophy

(2009) Ulrich Matzner et al.   MOLECULAR THERAPY

Metachromatic leukodystrophy: genetics, pathogenesis and therapeutic options

(2008) Volkmar Gieselmann   ACTA PAEDIATRICA

The Asialoglycoprotein Receptor Regulates Levels of Plasma Glycoproteins Terminating with Sialic Acid α2,6-Galactose

(2008) Lindsay M. Steirer et al.   JOURNAL OF BIOLOGICAL CHEMISTRY