Publications by authors named "Laura Adang"

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Critical Functional Domains in Pediatric Onset TUBB4A-Related Leukodystrophy: A Clinical and Caregiver's Perspective.
Francesco Gavazzi, Virali Patel, Jacqueline A Erler, Brittany Charsar, Ylenia Vaia, Laura A Adang

Pediatr Neurol· December 2025

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Exploration Into Lived Experiences of Multiple Sulfatase Deficiency-Affected Individuals and Their Families.
Francesco Gavazzi, Emily Yu, Zarrin Tashnim, Sarah Woidill, Anjana Sevagamoorthy, Laura A Adang

J Child Neurol· November 2025

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Functional ability profiles in beta-propeller protein-associated neurodegeneration (BPAN).
Francesco Gavazzi, Samuel R Pierce, Vanessa Smith, Eric Yang, Julie Skorup, Laura A Adang

Mol Genet Metab· November 2025

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Clinical Characterization of a Multicenter International Cohort of Patients With Aicardi-Goutières Syndrome Homozygous for the RNASEH2B:p.Ala177Thr Variant: Early Clinical Markers of Disease Severity.
Costanza Varesio, Davide Politano, Laura Adang, Elena Ballante, Roberta Battini

Pediatr Neurol· October 2025

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Experiences and Hope in Caregivers of Children With Aicardi Goutières Syndrome.
Francesco Gavazzi, Ashley Martin, Anjana Sevagamoorthy, Ylenia Vaia, Ariel Vincent, Laura A Adang

J Child Neurol· August 2025

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Reporting ABCD1 variants as actionable secondary findings on exome and genome sequencing.
Carlos A Dominguez Gonzalez, Nancy B Spinner, Rebecca C Ahrens-Nicklas, Lisa R Young, Laura A Voss, Laura Adang

Genet Med· July 2025

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Characterizing Diagnostic Delays in Metachromatic Leukodystrophy: A Real-World Data Approach.
Ali Mohajer, Anjana Sevagamoorthy, Karen Bean, Sylvia Mutua, Francis Pang, Laura Ann Adang

J Inherit Metab Dis· July 2025

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Key lessons from the first international treatment eligibility committee: the case of metachromatic leukodystrophy.
Daphne H Schoenmakers, Marije A B C Asbreuk, Tamara Martin, Mareen Datema, Shanice Beerepoot, Laura A Adang

Eur J Paediatr Neurol· July 2025

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Metachromatic Leukodystrophy: New Therapy Advancements and Emerging Research Directions.
Marije A B C Asbreuk, Daphne H Schoenmakers, Laura Ann Adang, Shanice Beerepoot, Caroline Bergner

Neurology· July 2025

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Clinically Important Endpoints in Individuals With Leukodystrophy: A Multisite Study.
Emma R Kotes, Sarah Woidill, Russell D'Aiello, Amina Khan, Jacob McCann, Laura Adang

Ann Child Neurol Soc· July 2025

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The natural history of variable subtypes in pediatric-onset TUBB4A-related leukodystrophy.
Francesco Gavazzi, Brittany Charsar, Eline Hamilton, Jacqueline A Erler, Virali Patel, Laura A Adang

Mol Genet Metab· March 2025

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Effective gene therapy for metachromatic leukodystrophy achieved with minimal lentiviral genomic integrations.
Lucas Tricoli, Sunetra Sase, Julia L Hacker, Vi Pham, Maxwell Chappell, Laura A Adang

Mol Ther Nucleic Acids· March 2025

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Current Advances and Challenges in Gene Therapies for Neurologic Disorders: A Review for the Clinician.
Giulia Stefania Porcari, John Warren Collyer, Laura Ann Adang, Deepa Soundara Rajan

Neurol Genet· February 2025

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Determination of Health Concepts in β-Propeller Protein-Associated Neurodegeneration.
Emma Kotes, Francesco Gavazzi, Sarah Woidill, Anjana Sevagamoorthy, Eric Yang, Laura A Adang

J Child Neurol· January 2025

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Characterization of gallbladder disease in metachromatic leukodystrophy across the lifespan.
Sylvia Mutua, Anjana Sevagamoorthy, Sarah Woidill, Paul J Orchard, Francesco Gavazzi, Laura A Adang

Mol Genet Metab· January 2025

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Bone marrow transplantation reverses metabolic alterations in multiple sulfatase deficiency: a case series.
Nishitha R Pillai, Ning Liu, Xiyuan Li, Xiqi Li, Rebecca Ahrens-Nicklas, Laura Adang

Commun Med (Lond)· January 2025

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Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of multiple sulfatase deficiency.
Vi Pham, Lucas Tricoli, Xinying Hong, Parith Wongkittichote, Carlo Castruccio Castracani, Laura A Adang

Mol Ther· November 2024

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Systemic complications of Aicardi Goutières syndrome using real-world data.
Isabella Peixoto de Barcelos, Amanda K Jan, Nicholson Modesti, Sarah Woidill, Francesco Gavazzi, Laura A Adang

Mol Genet Metab· October 2024

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Practical Approach to Longitudinal Neurologic Care of Adults With X-Linked Adrenoleukodystrophy and Adrenomyeloneuropathy.
Alexandra B Kornbluh, Aaron Baldwin, Ali Fatemi, Adeline Vanderver, Laura A Adang

Neurol Genet· October 2024

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Framework for Multistakeholder Patient Registries in the Field of Rare Diseases: Focus on Neurogenetic Diseases.
Daphne H Schoenmakers, Sibren van den Berg, Lonneke Timmers, Laura A Adang, Tobias Bäumer

Neurology· September 2024

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Developmental delay can precede neurologic regression in early onset metachromatic leukodystrophy.
Laura Ann Adang, Samuel Groeschel, Chloe Grzyb, Russell D'Aiello, Francesco Gavazzi

Mol Genet Metab· August 2024

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Consensus guidelines for the monitoring and management of metachromatic leukodystrophy in the United States.
Laura A Adang, Joshua L Bonkowsky, Jaap Jan Boelens, Eric Mallack, Rebecca Ahrens-Nicklas

Cytotherapy· July 2024

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Nonverbal Cognitive Skills in Children With Aicardi Goutières Syndrome.
Francesco Gavazzi, Ylenia Vaia, Sarah Woidill, Brielle Formanowski, Isabella Peixoto de Barcelos, Laura A Adang

Neurology· July 2024

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IFN-signaling gene expression as a diagnostic biomarker for monogenic interferonopathies.
Laura A Adang, Russell D'Aiello, Asako Takanohashi, Sarah Woidill, Francesco Gavazzi

JCI Insight· June 2024

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Metachromatic leukodystrophy: A story of hope woven from sorrow.
Laura Adang

Mol Ther· May 2024

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