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「"Heard JM "[Author]」の検索結果

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Availability, accessibility and delivery to patients of the 28 orphan medicines approved by the European Medicine Agency for hereditary metabolic diseases in the MetabERN network.

Research activity and capability in the European reference network MetabERN.

Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial.

Shotgun proteomics reveals possible mechanisms for cognitive impairment in Mucopolysaccharidosis I mice.

Oxidative stress is independent of inflammation in the neurodegenerative sanfilippo syndrome type B.

Heparan sulphate saccharides modify focal adhesions: Implication in mucopolysaccharidosis neuropathophysiology.

Removal of albumin and immunoglobulins from canine cerebrospinal fluid using depletion kits: a feasibility study.

Intracerebral administration of AAV rh.10 carrying human SGSH and SUMF1 cDNAs in children with MPSIIIA disease: results of a phase I/II trial.

GM130 gain-of-function induces cell pathology in a model of lysosomal storage disease.

Modeling neuronal defects associated with a lysosomal disorder using patient-derived induced pluripotent stem cells.

Delivery of therapeutic proteins from genetically-modified cells.

Incidence and natural history of mucopolysaccharidosis type III in France and comparison with United Kingdom and Greece.

Safe, efficient, and reproducible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes.

Neurotrophin 3 improves delayed reconstruction of sensory pathways after cervical dorsal root injury.

Storage problems in lysosomal diseases.

Storage vesicles in neurons are related to Golgi complex alterations in mucopolysaccharidosis IIIB.

GAP43 overexpression and enhanced neurite outgrowth in mucopolysaccharidosis type IIIB cortical neuron cultures.

Enhanced degradation of synaptophysin by the proteasome in mucopolysaccharidosis type IIIB.

Human alpha-iduronidase gene transfer mediated by adeno-associated virus types 1, 2, and 5 in the brain of nonhuman primates: vector diffusion and biodistribution.

Combination of microsurgery and gene therapy for spinal dorsal root injury repair.

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