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Infantile gangliosidoses: Mapping a timeline of clinical changesMOLECULAR GENETICS AND METABOLISMIF3.52017-06-0162OAAI
Identifying unmet needs for patients with Fabry disease through motivational interviewingMOLECULAR GENETICS AND METABOLISMIF3.52017-01-010PREAI
Infantile gangliosidoses: candidate outcome measures for future treatment trialsMOLECULAR GENETICS AND METABOLISMIF3.52017-01-010PREAI
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Characterization of oxidative stress and inflammation in type1 Gaucher disease-method evaluation and preliminary analysisMOLECULAR GENETICS AND METABOLISMIF3.52016-02-011PREAI
Characterizing infusion reactions to intravenous enzyme replacement therapy: establishing temporal relationships for effective management strategiesMOLECULAR GENETICS AND METABOLISMIF3.52016-02-010PREAI
Galsulfase (Naglazyme) after hematopoietic cell transplant for MPS VI (Maroteaux-Lamy syndrome)MOLECULAR GENETICS AND METABOLISMIF3.52016-02-010PREAI
Biomarkers of central nervous system inflammation in infantile and juvenile gangliosidosesMOLECULAR GENETICS AND METABOLISMIF3.52015-02-0147OAAI
Safety of caregiver administration of enzyme replacement therapy in the home settingMOLECULAR GENETICS AND METABOLISMIF3.52015-02-010PREAI
Prevalence of hypothyroidism in adult-onset Pompe diseaseMOLECULAR GENETICS AND METABOLISMIF3.52015-02-010PREAI
Biomarkers of central nervous system inflammation in infantile and juvenile gangliosidosesMOLECULAR GENETICS AND METABOLISMIF3.52015-02-010PREAI
Phenylketonuria Scientific Review Conference: State of the science and future research needs苯丙酮尿症科学审查会议: 科学状况和未来研究需求MOLECULAR GENETICS AND METABOLISMIF3.52014-06-01190OAAI
Evaluation and treatment of pain manifestations in Fabry disease: three case reportsMOLECULAR GENETICS AND METABOLISMIF3.52014-02-010OAAI
Eradication of inhibitors to enzyme replacement therapy in a Hunter syndrome patient using non-cytotoxic, non-immunosuppressing regimenMOLECULAR GENETICS AND METABOLISMIF3.52014-02-010PREAI
Metabolomic study of CSF and serum markers in infantile and juvenile gangliosidosis diseasesMOLECULAR GENETICS AND METABOLISMIF3.52014-02-010PREAI
TNF-α levels are increased in children with mucopolysaccharidosis types I, II, and VI treated with ERTMOLECULAR GENETICS AND METABOLISMIF3.52014-02-010PREAI
Combination oral therapy for lysosomal gangliosidoses using FDA approved medicationsMOLECULAR GENETICS AND METABOLISMIF3.52013-02-010OAAI
A longitudinal study of hexosaminidase deficiency (Tay-Sachs disease, Sandhoff disease)MOLECULAR GENETICS AND METABOLISMIF3.52013-02-010PREAI
The importance of precisely measured dietary management for patients on miglustat therapyMOLECULAR GENETICS AND METABOLISMIF3.52013-02-010PREAI