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Laura A Adang

Showing results (31-40 of 49) with videos related to

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Molecular Genetics and Metabolism|February 5, 2018
Complex care of individuals with multiple sulfatase deficiency: Clinical cases and consensus statementRebecca Ahrens-Nicklas, Lars Schlotawa, Andrea Ballabio, et al.
Pediatric Neurology|October 23, 2025
Critical Functional Domains in Pediatric Onset TUBB4A-Related Leukodystrophy: A Clinical and Caregiver's PerspectiveFrancesco Gavazzi, Virali Patel, Jacqueline A Erler, et al.
Molecular Genetics and Metabolism|September 27, 2024
Systemic complications of Aicardi Goutières syndrome using real-world dataIsabella Peixoto de Barcelos, Amanda K Jan, Nicholson Modesti, et al.
Molecular Genetics and Metabolism|July 5, 2022
Hematologic abnormalities in Aicardi Goutières SyndromeLaura A Adang, Francesco Gavazzi, Russell D'Aiello, et al.
Molecular Therapy. Nucleic Acids|April 2, 2025
Effective gene therapy for metachromatic leukodystrophy achieved with minimal lentiviral genomic integrationsLucas Tricoli, Sunetra Sase, Julia L Hacker, et al.
Pediatric Neurology|May 11, 2020
Phenotypic and Imaging Spectrum Associated With WDR45Laura A Adang, Amy Pizzino, Alka Malhotra, et al.
Molecular Genetics and Metabolism|May 23, 2026
Developmental trajectory of individuals with Pelizaeus-Merzbacher Disease (PMD)Anjana Sevagamoorthy, Sarah Woidill, Gabrielle Sudilovsky, et al.
Neurology|June 10, 2024
Nonverbal Cognitive Skills in Children With Aicardi Goutières SyndromeFrancesco Gavazzi, Ylenia Vaia, Sarah Woidill, et al.
European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society|March 30, 2024
Newborn screening in metachromatic leukodystrophy - European consensus-based recommendations on clinical managementLucia Laugwitz, Daphne H Schoenmakers, Laura A Adang, et al.
Molecular Genetics and Metabolism|February 11, 2026
A novel patient-Centered approach to clinical trial readiness in rare diseases: Application in Aicardi-Goutières Syndrome (AGS)Anjana Sevagamoorthy, Francesco Gavazzi, Zarrin Tashnim, et al.
Pageof 5

Showing results (31-40 of 49) with videos related to

Sort By:
Pageof 5
Molecular Genetics and Metabolism|February 5, 2018
Complex care of individuals with multiple sulfatase deficiency: Clinical cases and consensus statementRebecca Ahrens-Nicklas, Lars Schlotawa, Andrea Ballabio, et al.
Pediatric Neurology|October 23, 2025
Critical Functional Domains in Pediatric Onset TUBB4A-Related Leukodystrophy: A Clinical and Caregiver's PerspectiveFrancesco Gavazzi, Virali Patel, Jacqueline A Erler, et al.
Molecular Genetics and Metabolism|September 27, 2024
Systemic complications of Aicardi Goutières syndrome using real-world dataIsabella Peixoto de Barcelos, Amanda K Jan, Nicholson Modesti, et al.
Molecular Genetics and Metabolism|July 5, 2022
Hematologic abnormalities in Aicardi Goutières SyndromeLaura A Adang, Francesco Gavazzi, Russell D'Aiello, et al.
Molecular Therapy. Nucleic Acids|April 2, 2025
Effective gene therapy for metachromatic leukodystrophy achieved with minimal lentiviral genomic integrationsLucas Tricoli, Sunetra Sase, Julia L Hacker, et al.
Pediatric Neurology|May 11, 2020
Phenotypic and Imaging Spectrum Associated With WDR45Laura A Adang, Amy Pizzino, Alka Malhotra, et al.
Molecular Genetics and Metabolism|May 23, 2026
Developmental trajectory of individuals with Pelizaeus-Merzbacher Disease (PMD)Anjana Sevagamoorthy, Sarah Woidill, Gabrielle Sudilovsky, et al.
Neurology|June 10, 2024
Nonverbal Cognitive Skills in Children With Aicardi Goutières SyndromeFrancesco Gavazzi, Ylenia Vaia, Sarah Woidill, et al.
European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society|March 30, 2024
Newborn screening in metachromatic leukodystrophy - European consensus-based recommendations on clinical managementLucia Laugwitz, Daphne H Schoenmakers, Laura A Adang, et al.
Molecular Genetics and Metabolism|February 11, 2026
A novel patient-Centered approach to clinical trial readiness in rare diseases: Application in Aicardi-Goutières Syndrome (AGS)Anjana Sevagamoorthy, Francesco Gavazzi, Zarrin Tashnim, et al.
Pageof 5