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A prospective natural history study of Krabbe disease in a patient cohort with onset between 6 months and 3 years of life

Orphanet journal of rare diseases, 2018-08, Vol.13 (1), p.126-126, Article 126

2018

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Early treatment is associated with improved cognition in Hurler syndrome

Annals of neurology, 2014-11, Vol.76 (5), p.747-753

2014

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Developmental outcomes of cord blood transplantation for Krabbe disease: A 15-year study

Neurology, 2017-09, Vol.89 (13), p.1365

2017

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Long-term neurodevelopmental outcomes of hematopoietic stem cell transplantation for late-infantile Krabbe disease

Blood, 2021-04, Vol.137 (13), p.1719-1730

2021

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Early disease progression of Hurler syndrome

Orphanet journal of rare diseases, 2017-02, Vol.12 (1), p.32-32, Article 32

2017

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Trajectories of Early Brain Volume Development in Fragile X Syndrome and Autism

Journal of the American Academy of Child and Adolescent Psychiatry, 2012-09, Vol.51 (9), p.921-933

2012

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Hyporesponsiveness to social and nonsocial sensory stimuli in children with autism, children with developmental delays, and typically developing children

Development and psychopathology, 2013-05, Vol.25 (2), p.307-320

2013

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Natural progression of neurological disease in mucopolysaccharidosis type II

Pediatrics (Evanston), 2011-05, Vol.127 (5), p.e1258

2011

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To What Extent Do Joint Attention, Imitation, and Object Play Behaviors in Infancy Predict Later Communication and Intellectual Functioning in ASD?

Journal of autism and developmental disorders, 2012-06, Vol.42 (6), p.1064-1074

2012

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Clinical management of Krabbe disease

Journal of neuroscience research, 2016-11, Vol.94 (11), p.1118-1125

2016

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Sensory Experiences Questionnaire: discriminating sensory features in young children with autism, developmental delays, and typical development

Journal of child psychology and psychiatry, 2006-06, Vol.47 (6), p.591-601

2006

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Early progression of Krabbe disease in patients with symptom onset between 0 and 5 months

Orphanet journal of rare diseases, 2019-02, Vol.14 (1), p.46-46, Article 46

2019

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Trajectories of aggression from toddlerhood to age 9 predict academic and social functioning through age 12

Journal of child psychology and psychiatry, 2006-08, Vol.47 (8), p.791-800

2006

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Sensory features and repetitive behaviors in children with autism and developmental delays

Autism research, 2010-04, Vol.3 (2), p.78-87

2010

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Transplantation of Umbilical-Cord Blood in Babies with Infantile Krabbe's Disease

The New England journal of medicine, 2005-05, Vol.352 (20), p.2069-2081

2005

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Thickening of the cauda equina roots: a common finding in Krabbe disease

European radiology, 2016-10, Vol.26 (10), p.3377-3382

2016

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The Comprehensive Language Approach to Early Literacy: The Interrelationships Among Vocabulary, Phonological Sensitivity, and Print Knowledge Among Preschool-Aged Children

Journal of educational psychology, 2003-09, Vol.95 (3), p.465-481

2003

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Early Clinical Markers of Central Nervous System Involvement in Mucopolysaccharidosis Type II

The Journal of pediatrics, 2011-08, Vol.159 (2), p.320-326.e2

2011

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Development of a newborn screening tool based on bivariate normal limits: using psychosine and galactocerebrosidase determination on dried blood spots to predict Krabbe disease

Genetics in medicine, 2019-07, Vol.21 (7), p.1644-1651

2019

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O30: REKLAIM: A first in human phase Ib clinical trial of FBX-101 (AAVrh10.GALC) intravenously administered after UCBT for infantile Krabbe disease

Genetics in Medicine Open, 2024, Vol.2, p.100875, Article 100875

2024

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Cortical Gray and White Brain Tissue Volume in Adolescents and Adults with Autism

Biological psychiatry (1969), 2006, Vol.59 (1), p.1-6

2006

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Development of a mouse test for repetitive, restricted behaviors: Relevance to autism

Behavioural brain research, 2008-03, Vol.188 (1), p.178-194

2008

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The Emergence of Effortful Control in Young Boys with Fragile X Syndrome

American journal on intellectual and developmental disabilities, 2018-03, Vol.123 (2), p.89-191

2018

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A staging system for infantile Krabbe disease to predict outcome after unrelated umbilical cord blood transplantation

Pediatrics (Evanston), 2006-09, Vol.118 (3), p.e879

2006

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P014: First-in-human phase 1/2 trial of intravenous FBX-101 with cord blood transplantation increases GALC, brain and motor development in infantile Krabbe

Genetics in Medicine Open, 2023, Vol.1 (1), p.100024, Article 100024

2023

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A prospective natural history study of Krabbe disease in a patient cohort with onset between 6 months and 3 years of life

Early treatment is associated with improved cognition in Hurler syndrome

Developmental outcomes of cord blood transplantation for Krabbe disease: A 15-year study

Long-term neurodevelopmental outcomes of hematopoietic stem cell transplantation for late-infantile Krabbe disease

Early disease progression of Hurler syndrome

Trajectories of Early Brain Volume Development in Fragile X Syndrome and Autism

Hyporesponsiveness to social and nonsocial sensory stimuli in children with autism, children with developmental delays, and typically developing children

Natural progression of neurological disease in mucopolysaccharidosis type II

To What Extent Do Joint Attention, Imitation, and Object Play Behaviors in Infancy Predict Later Communication and Intellectual Functioning in ASD?

Clinical management of Krabbe disease

Sensory Experiences Questionnaire: discriminating sensory features in young children with autism, developmental delays, and typical development

Early progression of Krabbe disease in patients with symptom onset between 0 and 5 months

Trajectories of aggression from toddlerhood to age 9 predict academic and social functioning through age 12

Sensory features and repetitive behaviors in children with autism and developmental delays

Transplantation of Umbilical-Cord Blood in Babies with Infantile Krabbe's Disease

Thickening of the cauda equina roots: a common finding in Krabbe disease

The Comprehensive Language Approach to Early Literacy: The Interrelationships Among Vocabulary, Phonological Sensitivity, and Print Knowledge Among Preschool-Aged Children

Early Clinical Markers of Central Nervous System Involvement in Mucopolysaccharidosis Type II

Development of a newborn screening tool based on bivariate normal limits: using psychosine and galactocerebrosidase determination on dried blood spots to predict Krabbe disease

O30: REKLAIM: A first in human phase Ib clinical trial of FBX-101 (AAVrh10.GALC) intravenously administered after UCBT for infantile Krabbe disease

Cortical Gray and White Brain Tissue Volume in Adolescents and Adults with Autism

Development of a mouse test for repetitive, restricted behaviors: Relevance to autism

The Emergence of Effortful Control in Young Boys with Fragile X Syndrome

A staging system for infantile Krabbe disease to predict outcome after unrelated umbilical cord blood transplantation

P014: First-in-human phase 1/2 trial of intravenous FBX-101 with cord blood transplantation increases GALC, brain and motor development in infantile Krabbe

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