Multisystemic Manifestations of Underlying Metabolic Diseases

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Faculty

Barbara K. Burton, MD

Professor of Pediatrics
Northwestern University Feinberg School of Medicine
Attending Physician
Division of Genetics, Birth Defects and Metabolism
Ann and Robert H. Lurie Children's Hospital
Chicago, Illinois

Disclosures

Disclosure: Barbara K. Burton, MD, has the following relevant financial relationships:
Served as an advisor or consultant for: Aeglea BioTherapeutics; Alexion Pharmaceuticals, Inc.; Applied Therapeutics; BioMarin Pharmaceutical Inc; Denali Therapeutics; Genzyme; Horizon Pharma; JCR Pharmaceuticals; Moderna, Inc.; Orchard Therapeutics; Passage Bio; Sio Gene Therapies; Takeda; Ultragenyx
Served as a speaker or a member of a speakers bureau for: BioMarin Pharmaceutical Inc; Takeda
Received grants for clinical research from: BioMarin Pharmaceutical Inc; Denali Therapeutics; Homology Medicines; JCR Pharmaceuticals; Sangamo BioSciences, Inc.; Takeda; Ultragenyx

Roberto Giugliani, MD, PhD

Department of Genetics/UFRGS
Medical Genetics Service/HCPA
DASA, Casa dos Raros
Porto Alegre, Brazil

Disclosures

Disclosure: Roberto Giugliani, MD, PhD, has the following relevant financial relationships:
Served as an advisor or consultant for: Abeona Therapeutics Inc.; Amicus Therapeutics; Azafaros; Idorsia Pharmaceuticals Ltd; Inventiva Pharma; Janssen; JCR Pharma; RegenxBio; Sanofi; Sigilon Therapeutics; Takeda
Served as a speaker or a member of a speakers bureau for: Amicus Therapeutics; BioMarin Pharmaceutical Inc; Janssen; JCR Pharma; Novartis; PTC Therapeutics; RegenxBio; Sanofi; Takeda; Ultragenyx
Received grants for clinical research from: PTC
Served as a contracted researcher for the following ineligible companies: Allievex; Avrobio; Azafaros; BioMarin Pharmaceutical Inc; Idorsia Pharmaceuticals Ltd; JCR Pharmaceuticals; Lysogene; Paradigm; Passage Bio; PTC Therapeutics; RegenxBio; Sanofi; Sigilon Therapeutics; Takeda; Ultragenyx

Martha Solano, MD

Neuropediatrician
Principal investigator
Fundacion Cardioinfantil
Bogotá, Colombia

Disclosures

Disclosure: Martha Solano, MD, has no relevant financial relationships.

Klane K. White, MD, MSc

Director
Skeletal Health and Dysplasia Program
Medical Director
Orthopaedic Research
Professor
Orthopedics and Sports Medicine
Seattle Children's Hospital
University of Washington
Seattle, Washington, United States

Disclosures

Disclosure: Klane K. White, MD, MSc, has the following relevant financial relationships:
Served as an advisor or consultant for: BioMarin Pharmaceutical Inc
Served as a speaker or a member of a speakers bureau for: BioMarin Pharmaceutical Inc
Received grants for clinical research from: BioMarin Pharmaceutical Inc; Pfizer, Inc.; TherAchon; Ultragenyx

Editor

Rita Moreira da Silva, PhD, MA, PharmD

Medical Education Director, WebMD Global, LLC

Disclosures

Disclosure: Rita Moreira da Silva, PhD, MA, PharmD, has no relevant financial relationships.

Compliance Reviewer

Leigh Schmidt, MSN, RN, CMSRN, CNE, CHCP

Associate Director, Accreditation and Compliance

Disclosures

Disclosure: Leigh Schmidt, MSN, RN, CMSRN, CNE, CHCP, has no relevant financial relationships.

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Disclaimer

The educational activity presented above may involve simulated case-based scenarios. The patients depicted in these scenarios are fictitious and no association with any actual patient is intended or should be inferred.

The material presented here does not necessarily reflect the views of WebMD Global, LLC or companies that support educational programming on medscape.org. A qualified healthcare professional should be consulted before using any therapeutic product discussed. Readers should verify all information and data before treating patients or employing any therapies described in this educational activity.

Faculty and Disclosures

Faculty

Barbara K. Burton, MD

Roberto Giugliani, MD, PhD

Martha Solano, MD

Klane K. White, MD, MSc

Editor

Rita Moreira da Silva, PhD, MA, PharmD

Compliance Reviewer

Leigh Schmidt, MSN, RN, CMSRN, CNE, CHCP

Multisystemic Manifestations of Underlying Metabolic Diseases