The association between tooth brushing during lunchtime and obesity in South Korean adolescentsSCIENTIFIC REPORTSIF3.92024-09-050OAAI
Membrane association of active genes organizes the chloroplast nucleoid structurePROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICAIF9.12024-07-050PRE
First in-human, intracisternal dosing of RGX-111, an investigational AAV gene therapy, for a 21-month-old child with mucopolysaccharidosis type I (MPS I): 3.5 year follow-upMOLECULAR GENETICS AND METABOLISMIF3.52024-02-011PRE
The relationship between the expanded neuronal ceroid lipofuscinosis 2 (CLN2) clinical rating scale for motor function (CLN2 CRS-MX) and GAITRite® parametersMOLECULAR GENETICS AND METABOLISMIF3.52023-02-010PRE
Quantifying and modelling disease progression trajectory for natural history of MPS IIMOLECULAR GENETICS AND METABOLISMIF3.52023-02-010PRE
Daily living skills on the Vineland Adaptive Behavioral Scale version 2 (VABS-II) in neuronopathic mucopolysaccharidosis type II (MPSMOLECULAR GENETICS AND METABOLISMIF3.52023-02-010PRE
RGX-111 gene therapy for the treatment of severe mucopolysaccharidosis type I (MPS I): Interim analysis of data from the first in-human studyMOLECULAR GENETICS AND METABOLISMIF3.52022-02-010PRE
Beyond the normative data: Understanding the Mullen Scales of Early Learning (MSEL)MOLECULAR GENETICS AND METABOLISMIF3.52022-02-012PRE
Beyond the normative data: Understanding the Bayley Scales of Infant Development version 3 (BSID-III)MOLECULAR GENETICS AND METABOLISMIF3.52022-02-010PRE
Identification of a biomarker that differentiates neuronopathic forms of MPS I and MPS IIMOLECULAR GENETICS AND METABOLISMIF3.52022-02-012PRE
RGX-121 gene therapy for the treatment of severe mucopolysaccharidosis type II (MPS II): Interim analysis of data from the first in-human studyMOLECULAR GENETICS AND METABOLISMIF3.52022-02-011PRE
The expanded neuronal ceroid lipofuscinosis 2 (CLN2) clinical rating scale for motor and language function: Development and inter-rater reliabilityMOLECULAR GENETICS AND METABOLISMIF3.52022-02-010PRE
First in-human intracisternal dosing of RGX-111 (adeno-associated virus 9/human α-L-iduronidase) for a 20-month-old child with mucopolysaccharidosis type I (MPS I): 1 year follow-upMOLECULAR GENETICS AND METABOLISMIF3.52021-02-010PREAI
Clinical and numerical presentation of neurocognitive assessments for MPS II patients using the Bayley Scales of Infant Development- version 3 (BSID-III)MOLECULAR GENETICS AND METABOLISMIF3.52021-02-010PREAI
RGX-121 gene therapy for severe mucopolysaccharidosis type II (MPS II): Interim results of an ongoing first in human trialMOLECULAR GENETICS AND METABOLISMIF3.52021-02-012PREAI