A Systematic Literature Review on the Global Status of Newborn Screening for Mucopolysaccharidosis IIINTERNATIONAL JOURNAL OF NEONATAL SCREENINGIF42024-10-100OAAI
Clinical investigator perspectives on patient outcomes in children with neuronopathic mucopolysaccharidosis II during intrathecal idursulfase-IT treatmentORPHANET JOURNAL OF RARE DISEASESIF3.52024-04-120OAAI
Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis IIORPHANET JOURNAL OF RARE DISEASESIF3.52024-03-100OAAI
A post hoc analysis of Projected Retained Ability Scores (PRAS) for the longitudinal assessment of cognitive functioning in patients with neuronopathic mucopolysaccharidosis II receiving intrathecal idursulfase-ITORPHANET JOURNAL OF RARE DISEASESIF3.52023-11-021OAAI
Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study (vol 137, pg 127, 2022)MOLECULAR GENETICS AND METABOLISMIF3.52023-11-010OAAI
Cognitive and adaptive behaviors associated with disease severity and genotype in patients with mucopolysaccharidosis IIMOLECULAR GENETICS AND METABOLISMIF3.52023-11-011OAAI
Long-term open-label extension study of the safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II (vol 137, pg 22, 2022)MOLECULAR GENETICS AND METABOLISMIF3.52023-11-010OAAI
A retrospective chart review of supportive care in patients with mucopolysaccharidosis type II in the United StatesMOLECULAR GENETICS AND METABOLISMIF3.52023-02-010PRE
Long-term open-label extension study of the safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis IIMOLECULAR GENETICS AND METABOLISMIF3.52022-09-018OAAI
Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized studyMOLECULAR GENETICS AND METABOLISMIF3.52022-09-0124PRE
Clinical investigator perspectives on the effects of intrathecal idursulfase-IT treatment in children with neuronopathic mucopolysaccharidosis type IIMOLECULAR GENETICS AND METABOLISMIF3.52022-02-010PRE
Novel insights into mucopolysaccharidosis type II based on an analysis of genetic variants in 763 patientsMOLECULAR GENETICS AND METABOLISMIF3.52022-02-010PRE
Analysis of cognitive ability and adaptive behavior assessment tools used in an observational study of patients with mucopolysaccharidosis IIORPHANET JOURNAL OF RARE DISEASESIF3.52021-12-048OAAI
Assessment of cognitive development in patients with neuronopathic mucopolysaccharidosis type II treated with intrathecal idursulfase-IT using Projected Retained Ability Score (PRAS): A post hoc analysisMOLECULAR GENETICS AND METABOLISMIF3.52021-02-010PREAI
Long-term safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis type II: 2-year results from a phase 2/3 extension studyMOLECULAR GENETICS AND METABOLISMIF3.52021-02-010PREAI
Comparison of cognitive function in siblings with neuronopathic mucopolysaccharidosis type II: Evaluation of early treatment with intravenous idursulfase and intrathecal idursulfase-ITMOLECULAR GENETICS AND METABOLISMIF3.52021-02-010PREAI
Caregiver experiences of intrathecal idursulfase-IT treatment in pediatric patients with neuronopathic mucopolysaccharidosis type IIMOLECULAR GENETICS AND METABOLISMIF3.52021-02-010PREAI
Single-arm, open-label, phase 2/3 substudy and extension evaluating safety and efficacy of intrathecal idursulfase-IT in patients younger than 3 years old with neuronopathic mucopolysaccharidosis typeMOLECULAR GENETICS AND METABOLISMIF3.52021-02-010PREAI
Clinical characteristics and health care resource utilization for patients with mucopolysaccharidosis type II in the US: A retrospective chart reviewMOLECULAR GENETICS AND METABOLISMIF3.52020-02-010PREAI
Projected Retained Ability Score (PRAS): A New Methodology for Quantifying Absolute Change in Norm-Based Psychological Test Scores Over TimeASSESSMENTIF3.42019-09-1314OAAI
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