First-in-human intracisternal dosing of RGX-111 in severe MPS I is well-tolerated and generates sustained neurodevelopment without HSCTRGX-111在严重MPS I中的首次人体脑池内给药耐受性良好且无需HSCT即可产生持续神经发育Molecular TherapyIF122026-04-100OAAI
Audiology assessment of participants in CAMPSIITE®, a phase I/II/ III study of investigational RGX-121 in neuronopathic MPS IIMOLECULAR GENETICS AND METABOLISMIF3.52025-02-010PRE
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OCT Biomarkers in Ocular CLN2 Disease in Patients Treated With Intraventricular Enzyme Replacement TherapyINVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCEIF4.72024-07-300OAAI
CAMPSIITET Phase I/II/III: An interim clinical study update of RGX-121, an investigational gene therapy for the treatment of neuronopathic mucopolysaccharidosis type II (MPS II)MOLECULAR GENETICS AND METABOLISMIF3.52024-04-010PRE
RGX-381: Interim results from the first-in-human clinical trial of an investigational gene therapy for the treatment of ocular manifestations of CLN2 Batten diseaseMOLECULAR GENETICS AND METABOLISMIF3.52024-02-010PRE
CAMPSIITETM phase I/II/III: An interim clinical study update of RGX-121, an investigational gene therapy for the treatment of neuronopathic mucopolysaccharidosis type II (MPS II)MOLECULAR GENETICS AND METABOLISMIF3.52024-02-011PRE
Audiology assessment of participants in CAMPSIITET, a phase I/II/ III study of RGX-121 in neuronopathic MPS IIMOLECULAR GENETICS AND METABOLISMIF3.52024-02-010PRE
Interim results from the first-in-human intracisternal dosing of RGX-181 investigational AAV9 gene therapy in a child with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2)MOLECULAR GENETICS AND METABOLISMIF3.52024-02-010PRE
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 METABOLISMIF3.52023-03-010PRE
RGX-121 gene therapy for the treatment of neuronopathic mucopolysaccharidosis type II (MPS II): Interim analysis of data from the first in human studyMOLECULAR GENETICS AND METABOLISMIF3.52023-02-010PRE
Characterization of retinal degeneration phenotype in classic CLN2 disease using OCT biomarkers and an in vitro retinal modelMOLECULAR 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 METABOLISMIF3.52023-02-010PRE
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
Loss of visual function associated with photoreceptor degeneration in CLN2 diseaseMOLECULAR 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
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
Intracisternal administration of AAV9 gene therapies to target the central nervous systemMOLECULAR GENETICS AND METABOLISMIF3.52021-02-010PREAI
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
RGX-121 gene therapy for severe mucopolysaccharidosis type II (MPS II): A clinical program to address central nervous system manifestationsMOLECULAR GENETICS AND METABOLISMIF3.52021-02-012PREAI
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