Advances in the treatment of lysosomal storage disease

Long-Term Cognitive and Functional Outcomes in Children with Mucopolysaccharidosis (MPS)-IH (Hurler Syndrome) Treated with Hematopoietic Cell Transplantation.

JIMD reports·2016

Mucopolysaccharidosis type I.

Pediatric endocrinology reviews : PER·2014

Developmental outcome post allogenic bone marrow transplant for Niemann Pick Type C2.

Molecular genetics and metabolism·2012

Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme®) therapy.

Journal of inherited metabolic disease·2012

Development of a suspicion index to aid diagnosis of Niemann-Pick disease type C.

Neurology·2012

Female mucopolysaccharidosis IIIA mice exhibit hyperactivity and a reduced sense of danger in the open field test.

PloS one·2011

Caring for a child with cerebral palsy in rural Malawi: A mixed-methods study of caregiver experiences and support needs.

Developmental medicine and child neurology·2026

Vertigo and dizziness syndromes in children and adolescents: Clinical presentation and diagnostic pathway.

Developmental medicine and child neurology·2026

International consensus recommendations for identifying malnutrition in children with neurological impairment.

Developmental medicine and child neurology·2026

Pressure garment therapy and bimanual upper limb function in children with unilateral cerebral palsy: A randomized clinical trial.

Developmental medicine and child neurology·2026

Combined dorsal and ventral rhizotomy in non-ambulatory individuals with mixed spastic bilateral cerebral palsy: A prospective registry analysis.

Developmental medicine and child neurology·2026

Physical therapy interventions for children and adolescents with myelomeningocele: A systematic review.

Developmental medicine and child neurology·2026

A sensitive Interactome-based approach to characterize drug activity in living human cells.

The Journal of biological chemistry·2026

Conformational flexibility governs phosphate-induced solubility enhancement: a comparative study of cimetidine and famotidine by pH-dependent dissolution, NMR titration, DOSY, and DFT analysis.

European journal of pharmaceutics and biopharmaceutics : official journal of Arbeitsgemeinschaft fur Pharmazeutische Verfahrenstechnik e.V·2026

Making Modern Diabetes Medications Affordable and Accessible: Lessons From India for Other Countries.

Diabetes care·2026

Repurposing Anticancer Drugs in Parkinson's Treatment: Molecular Pathways Driving Neuroprotection and Therapeutic Advancement.

Molecular neurobiology·2026

Changes in peripheral inflammatory cytokines and clinical outcomes before and after deep brain stimulation in patients with Parkinson's disease with or without type 2 diabetes mellitus: an observational study.

Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology·2026

Inhalable Dry Powders of Antimicrobial Agents in Respiratory Diseases.

Lung·2026

Related Experiment Videos

Advances in the treatment of lysosomal storage disease

Related Concept Videos

Long-Term Cognitive and Functional Outcomes in Children with Mucopolysaccharidosis (MPS)-IH (Hurler Syndrome) Treated with Hematopoietic Cell Transplantation.

Mucopolysaccharidosis type I.

Developmental outcome post allogenic bone marrow transplant for Niemann Pick Type C2.

Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme®) therapy.

Development of a suspicion index to aid diagnosis of Niemann-Pick disease type C.

Female mucopolysaccharidosis IIIA mice exhibit hyperactivity and a reduced sense of danger in the open field test.

Caring for a child with cerebral palsy in rural Malawi: A mixed-methods study of caregiver experiences and support needs.

Vertigo and dizziness syndromes in children and adolescents: Clinical presentation and diagnostic pathway.

International consensus recommendations for identifying malnutrition in children with neurological impairment.

Pressure garment therapy and bimanual upper limb function in children with unilateral cerebral palsy: A randomized clinical trial.

Combined dorsal and ventral rhizotomy in non-ambulatory individuals with mixed spastic bilateral cerebral palsy: A prospective registry analysis.

Physical therapy interventions for children and adolescents with myelomeningocele: A systematic review.

A sensitive Interactome-based approach to characterize drug activity in living human cells.

Conformational flexibility governs phosphate-induced solubility enhancement: a comparative study of cimetidine and famotidine by pH-dependent dissolution, NMR titration, DOSY, and DFT analysis.

Making Modern Diabetes Medications Affordable and Accessible: Lessons From India for Other Countries.

Repurposing Anticancer Drugs in Parkinson's Treatment: Molecular Pathways Driving Neuroprotection and Therapeutic Advancement.

Changes in peripheral inflammatory cytokines and clinical outcomes before and after deep brain stimulation in patients with Parkinson's disease with or without type 2 diabetes mellitus: an observational study.

Inhalable Dry Powders of Antimicrobial Agents in Respiratory Diseases.

Related Experiment Videos

Advances in the treatment of lysosomal storage disease

Related Concept Videos

Related Articles