Pages that link to "Q39773493"
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The following pages link to Characterization of pre- and post-treatment pathology after enzyme replacement therapy for Pompe disease (Q39773493):
Displaying 50 items.
- Autophagy in lysosomal storage disorders (Q24601671) (← links)
- Murine muscle cell models for Pompe disease and their use in studying therapeutic approaches (Q24647307) (← links)
- New insights into therapeutic options for Pompe disease (Q26851649) (← links)
- Autophagy and mitochondria in Pompe disease: nothing is so new as what has long been forgotten (Q27015911) (← links)
- Pathophysiological concepts in the congenital myopathies: blurring the boundaries, sharpening the focus (Q28087567) (← links)
- Hearing loss in Pompe disease revisited: results from a study of 24 children (Q30479184) (← links)
- Skeletal muscle quantitative nuclear magnetic resonance imaging follow-up of adult Pompe patients (Q30906462) (← links)
- Increased aortic stiffness and blood pressure in non-classic Pompe disease (Q33579864) (← links)
- Fiber Type Conversion by PGC-1α Activates Lysosomal and Autophagosomal Biogenesis in Both Unaffected and Pompe Skeletal Muscle (Q33778251) (← links)
- Pompe disease: from pathophysiology to therapy and back again (Q34054935) (← links)
- Autophagy in skeletal muscle: implications for Pompe disease (Q34170362) (← links)
- Role of autophagy in the pathogenesis of Pompe disease. (Q34171689) (← links)
- The pharmacological chaperone AT2220 increases recombinant human acid α-glucosidase uptake and glycogen reduction in a mouse model of Pompe disease (Q34345151) (← links)
- Differences in the predominance of lysosomal and autophagic pathologies between infants and adults with Pompe disease: implications for therapy (Q34349099) (← links)
- Allele copy number and underlying pathology are associated with subclinical severity in equine type 1 polysaccharide storage myopathy (PSSM1) (Q34364791) (← links)
- Peripheral nerve and neuromuscular junction pathology in Pompe disease (Q34920690) (← links)
- Myostatin and insulin-like growth factor I: potential therapeutic biomarkers for pompe disease (Q34964663) (← links)
- Identification and Characterization of Pharmacological Chaperones to Correct Enzyme Deficiencies in Lysosomal Storage Disorders (Q35007910) (← links)
- Pompe disease results in a Golgi-based glycosylation deficit in human induced pluripotent stem cell-derived cardiomyocytes. (Q35049500) (← links)
- β2 Agonists enhance the efficacy of simultaneous enzyme replacement therapy in murine Pompe disease (Q35690999) (← links)
- Postmortem Findings and Clinical Correlates in Individuals with Infantile-Onset Pompe Disease (Q35795394) (← links)
- Defects in calcium homeostasis and mitochondria can be reversed in Pompe disease (Q35853122) (← links)
- Liver and skin histopathology in adults with acid sphingomyelinase deficiency (Niemann-Pick disease type B) (Q36094972) (← links)
- Muscle MRI Findings in Childhood/Adult Onset Pompe Disease Correlate with Muscle Function. (Q36154874) (← links)
- Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning (Q36164927) (← links)
- Lack of robust satellite cell activation and muscle regeneration during the progression of Pompe disease (Q36223120) (← links)
- Optimization of a histopathological biomarker for sphingomyelin accumulation in acid sphingomyelinase deficiency (Q36285882) (← links)
- Biochemical evidence for superior correction of neuronal storage by chemically modified enzyme in murine mucopolysaccharidosis VII. (Q36342413) (← links)
- Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study (Q36459627) (← links)
- Acid alpha-glucosidase deficiency (Pompe disease). (Q36705466) (← links)
- Lysosomal Storage Diseases-Regulating Neurodegeneration (Q36771137) (← links)
- Effects of a higher dose of alglucosidase alfa on ventilator-free survival and motor outcome in classic infantile Pompe disease: an open-label single-center study (Q36853458) (← links)
- Pompe disease: current state of treatment modalities and animal models. (Q36934500) (← links)
- Skeletal muscle pathology of infantile Pompe disease during long-term enzyme replacement therapy (Q36953239) (← links)
- Suppression of autophagy in skeletal muscle uncovers the accumulation of ubiquitinated proteins and their potential role in muscle damage in Pompe disease (Q37089630) (← links)
- Clearance of Hepatic Sphingomyelin by Olipudase Alfa Is Associated With Improvement in Lipid Profiles in Acid Sphingomyelinase Deficiency (Q37180476) (← links)
- Mitochondrial Copy Number and D-Loop Variants in Pompe Patients. (Q37232026) (← links)
- A review of treatment of Pompe disease in infants (Q37290516) (← links)
- Pompe's disease (Q37302098) (← links)
- Lower Urinary Tract Symptoms and Incontinence in Children with Pompe Disease (Q37329236) (← links)
- Therapeutic approaches in glycogen storage disease type II/Pompe Disease. (Q37329535) (← links)
- Temporal neuropathologic and behavioral phenotype of 6neo/6neo Pompe disease mice (Q37345062) (← links)
- Biochemical and pharmacological characterization of different recombinant acid alpha-glucosidase preparations evaluated for the treatment of Pompe disease (Q37414571) (← links)
- Divergent clinical outcomes of alpha-glucosidase enzyme replacement therapy in two siblings with infantile-onset Pompe disease treated in the symptomatic or pre-symptomatic state (Q37433545) (← links)
- Modulation of mTOR signaling as a strategy for the treatment of Pompe disease (Q37672756) (← links)
- Sustained correction of motoneuron histopathology following intramuscular delivery of AAV in pompe mice. (Q37696025) (← links)
- Newborn screening for neuropathic lysosomal storage disorders. (Q37763610) (← links)
- Challenges in diagnosis and treatment of late-onset Pompe disease (Q37926444) (← links)
- Glycogen storage diseases: a brief review and update on clinical features, genetic abnormalities, pathologic features, and treatment (Q37930640) (← links)
- Enzyme replacement therapy for Pompe disease. (Q37946297) (← links)