Gross Motor Function in Pediatric Onset TUBB4A-Related Leukodystrophy: GMFM-88 Performance and Validation of GMFC-MLD in TUBB4A
- Francesco Gavazzi*
- , Virali Patel
- , Brittany Charsar
- , Allan Glanzman
- , Jacqueline Erler
- , Anjana Sevagamoorthy
- , Emma McKenzie
- , Tracy Kornafel
- , Elizabeth Ballance
- , Samuel R. Pierce
- , Michelle Teng
- , Brielle Formanowski
- , Sarah Woidill
- , Justine Shults
- , Evangeline Wassmer
- , Davide Tonduti
- , Francesca Magrinelli
- , Geneviève Bernard
- , Marjo Van Der Knaap
- , Nicole Wolf
*Corresponding author for this work
- Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA
- Department of Physical Therapy, Children's Hospital of Philadelphia, Philadelphia, PA, USA
- Synaptixbio Ltd, Oxfordshire, United Kingdom
- Division of Neonatology, Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA, USA
- Department of Biomedical and Clinical Sciences, L. Sacco University Hospital, Università degli Studi di Milano, Milan, Italy
- Department of Clinical and Movement Neurosciences, UCL Queen Square Institute of Neurology, University College London, London, United Kingdom
- Child Health and Human Development Program, Research Institute of the McGill University Health Centre, Montreal, QC, Canada
- Department of Integrative Neurophysiology, Center for Neurogenomics and Cognitive Research, Vrije Universiteit, Amsterdam, the Netherlands
- Department of Child Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers, and Amsterdam Neuroscience, Cellular & Molecular Mechanisms, Vrije Universiteit, Amsterdam, the Netherlands
- Department of Neurology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA
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