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Laura Adang

Has grant

Assistant Professor

Children's Hospital of Philadelphia

United States

Research Interests

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Recent Grants

Grant: Open

Patient-Centric Clinical Outcome Measures for Multiple Sulfatase Deficiency (MSD)

Open Date: 2025-09-03

Close Date: 2027-09-02

Grant: Open

Database Harmonization for Creation and Validation of Outcome Assessments and Prediction Tools for Metachromatic Leukodystrophy (MLD)

Open Date: 2025-06-01

Close Date: 2027-05-31

Grant: Open

Predoctoral Preparatory Program (P3) in Translational Research

Open Date: 2024-05-01

Close Date: 2027-04-30

Grant: Close

Clinical measures in Aicardi Goutieres Syndrome

Open Date: 2019-09-30

Close Date: 2025-08-31

Grant: Open

Global Leukodystrophy Initiative Clinical Trials Network (GLIA- CTN)

Open Date: 2019-09-30

Close Date: 2030-08-31

Articles (20)

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Collaborators (9)

Nishitha Pillai

University of Minnesota Twin Cities

UNITED STATES

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Ludger Schöls

Section head

Eberhard Karls Universität Tübingen

GERMANY

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Sarah Elsea

Professor

Baylor College of Medicine

UNITED STATES

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Troy C. Lund

University of Minnesota

UNITED STATES

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Laura Breda

Research Assistant professor

Children's Hospital of Philadelphia

UNITED STATES

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Julie B. Eisengart

Faculty, Associate Professor

University of Minnesota

UNITED STATES

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Lisa Young

Children's Hospital of Philadelphia

UNITED STATES

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Anjana Sevagamoorthy

Staff Scientist

Children's Hospital of Philadelphia

UNITED STATES

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Elena Ballante

University of Pavia

ITALY

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Laura Adang

Research Interests

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Recent Grants

Articles (20)

A novel patient-Centered approach to clinical trial readiness in rare diseases: Application in Aicardi-Goutie`res Syndrome (AGS)

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

Bone marrow transplantation reverses metabolic alterations in multiple sulfatase deficiency: a case series

Critical Functional Domains in Pediatric Onset TUBB4A-Related Leukodystrophy: A Clinical and Caregiver's Perspective.

Characterizing Diagnostic Delays in Metachromatic Leukodystrophy: A Real‐World Data Approach

Characterization of gallbladder disease in metachromatic leukodystrophy across the lifespan

Determination of Health Concepts in β-Propeller Protein-Associated Neurodegeneration

Experiences and Hope in Caregivers of Children With Aicardi Goutières Syndrome

Exploration Into Lived Experiences of Multiple Sulfatase Deficiency-Affected Individuals and Their Families

The natural history of variable subtypes in pediatric-onset<i> TUBB4A</i>-related leukodystrophy

Current Advances and Challenges in Gene Therapies for Neurologic Disorders

Effective gene therapy for metachromatic leukodystrophy achieved with minimal lentiviral genomic integrations

Exploring the net monetary benefit of implementing newborn screening for metachromatic leukodystrophy in California

Functional ability profiles in beta-propeller protein-associated neurodegeneration (BPAN)

Key lessons from the first international treatment eligibility committee: the case of metachromatic leukodystrophy

Metachromatic Leukodystrophy

Reporting<i> ABCD1</i> variants as actionable secondary findings on exome and genome sequencing

A phase 2 study assessing TAK-611150 mg intrathecal weekly in patients with late-infantile metachromatic leukodystrophy (SHP611-201; EMBOLDEN) compared to matched historical control data from children with late-infantile MLD (GLIA-MLD)

Biochemical signatures of disease severity in multiple sulfatase deficiency

Characterization of Fine Motor and Visual Motor Skills in Aicardi-Goutières Syndrome

Collaborators (9)

Nishitha Pillai

Ludger Schöls

Sarah Elsea

Troy C. Lund

Laura Breda

Julie B. Eisengart

Lisa Young

Anjana Sevagamoorthy

Elena Ballante

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