REGENXBIO Announces Presentations at the 17th Annual WORLDSymposium™ 2021
The oral presentations will be presented as follows:
Abstract Title: Comparative systemic and neurologic effectiveness of intravenous and intrathecal AAV9 delivered individually or combined in a murine model of mucopolysaccharidosis type I
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Abstract Title: Intracisternal administration of AAV9 gene therapies to target the central nervous system
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Abstract Title: RGX-121 gene therapy for severe mucopolysaccharidosis type II (MPS II): Interim results of an ongoing first in human trial
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The poster presentations will be presented as follows:
Abstract Title: Subretinal injection of RGX-381 to cynomolgus monkeys leads to supraphysiological levels of TPP1 in the eye (poster #24)
Presenter: Nicholas Buss, Ph.D., Senior Director, Preclinical Development at
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Abstract Title: Intracisternal administration of AAV9 gene therapies to target the central nervous system (poster #207)
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Abstract Title: First in-human intracisternal dosing of RGX-111 (adeno-associated virus 9/human α-Liduronidase) for a 20-month-old child with mucopolysaccharidosis type I (MPS I): 1 year follow-up (poster #264)
Presenter: Raymond Wang, M.D., Biochemical Genetics Specialist at
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Abstract Title: RGX-121 gene therapy for severe mucopolysaccharidosis type II (MPS II): A clinical program to address central nervous system manifestations (poster #45)
Presenter: Kirsten Cowley, Director, Clinical Operations at
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Abstract Title: Clinical and numerical presentation of neurocognitive assessments for MPS II patients using the Bayley Scales of
Presenter: Yoonjin Cho, Ph.D., Director, Biostatistics at
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Abstract Title: RGX-121 gene therapy for severe mucopolysaccharidosis type II (MPS II): Interim results of an ongoing first in human trial (poster #171)
Presenter: Marie-
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Abstract Title: Characterizing expressive language skills in children with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2): The caregiver perspective (poster #198)
Presenter: Dawn Phillips, Ph.D., Director, Clinical Scientist,
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Abstract Title: Characterizing visual function in children with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2): The caregiver perspective (poster #199)
Presenter: Dawn Phillips, Ph.D., Director, Clinical Scientist,
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Abstract Title: New research examines the evolution of data sharing practices in natural history studies and patient registries among patient advocate leaders, industry and academia (poster #LB-12)
Presenter: Vivian Fernandez, Senior Director of Patient Advocacy at
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