Mucopolysaccharidosis I

1 paper

Animals1

Trial sponsors

Genzyme
Company1 trialPhase 1/27 people
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
1 trialPhase 2/32 people

Latest

1 in 12 months

Journal of Inherited Metabolic Disease29 Apr

Short-Term Oral Spermidine Supplementation Modifies Aspects of Neurodegenerative Disease in Flies and Mice With MPS III

Experiments in fliesSpermidine

Neural Regeneration Research2 May

Short-lived Niemann-Pick type C mice with accelerated brain aging as a novel model for Alzheimer's disease research

Experiments in miceMechanisms

The Ocular Surface12 Jan

Self-complementary AAV vector therapy for treating corneal cloudiness of mucopolysaccharidosis type VII (MPS VII)

Experiments in miceInterventions

Gene Therapy26 Apr

Significantly increased lifespan and improved behavioral performances by rAAV gene delivery in adult mucopolysaccharidosis IIIB mice

Experiments in miceInterventions

Molecular Therapy19 Apr

Genetic correction of the fetal brain increases the lifespan of mice with the severe multisystemic disease mucopolysaccharidosis type VII

Experiments in miceInterventions

Blood1 Dec

Increased life span and correction of metabolic defects in murine mucopolysaccharidosis type VII after syngeneic bone marrow transplantation

Experiments in miceInterventions