MPS Multidisciplinary Center
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Metabolic Disorders Referrals
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CHOC and Harbor-UCLA Pediatrics together offer the only place in the Western United States for children to receive comprehensive and coordinated treatment for a rare disease called mucopolysaccharidosis (MPS). In a single visit, patients and their families will see a geneticist, neurologist, endocrinologist, orthopaedic surgeon, and cardiologist, all of whom have expertise to collectively manage MPS and its effects on different areas of the body. Because patients with MPS should be continually checked for complications from the disease, we follow families closely to coordinate regular visits and ensure all of their needs are being met.
Learn more about the CHOC Metabolic Disorders Program
Comprehensive Treatment, All in One Place
At the CHOC-Harbor UCLA MPS Multidisciplinary Center, we offer:
- A comprehensive team of specialists who have expertise in treating children with MPS
- A single care setting for all diagnostic testing and treatment planning, for the convenience of families
- Close coordination with your child’s referring pediatrician and local subspecialists
- A holistic approach to treatment: we keep your child’s current and future health in mind and prepare for meaningful survivorship into adulthood
- The opportunity for families to participate in clinical and scientific research as we work to understand MPS and discover new and better treatments
- Connection with support from a nationwide MPS patient advocacy group, the National MPS Society.
On the Leading Edge of Research
The CHOC MPS Multidisciplinary Center team is actively involved in research to further our understanding of MPS and discover the latest treatments and protocols for this disease. Our patients may be invited to participate in clinical trials and studies to try alternate methods of care. Our current research includes:
Clinical Trials
- A Randomized, Placebo-Controlled, Blind-Start, Single-Crossover Phase 3 Study to Assess the Efficacy and Safety of UX003 rhGUS Enzyme Replacement Therapy in Patients with MPS 7 (Ultragenyx Pharmaceutical)
- A Phase 1 Safety and Dose-Finding Study of a Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) Fusion Protein, AGT-181 in Adult Patients with Mucopolysaccharidosis I (MPS I; Armagen)
- A Phase 1 Safety and Dose-Finding Study of a Human Insulin Receptor Monoclonal Antibody-Human Iduronate 2-Sulfatase (IDS) Fusion Protein, AGT-182 in Adult with Mucopolysaccharidosis II (MPS II, Hunter Syndrome; Armagen)
- A Pilot Study of the Effect of Adalimumab on Physical Function and Musculoskeletal Disease in Mucopolysaccharidosis Types I, II and VI (Investigator Initiated).
Clinical/Translational Research
- Assessment of cardiovascular disease natural history of the Mucopolysaccharidosis Type I Mouse model
- Carotid Structure and Function in MPS Types I, II, III and VI
- Manifestations of Cardiovascular Disease in Morquio A Syndrome
- Intraarticular Gene Therapy for Canine Mucopolysaccharidosis Type I Joint Disease
- Longitudinal Study of Bone and Endocrine Disease in Children with MPS I, II and VI: A Multicenter Study of the Lysosomal Disease Network
- Longitudinal studies of brain structure and function in MPS disorders.
Registry Studies
- MPS I Registry
- Hunter Outcome Registry
- Morquio A Registry Study.
The MPS Team
Dr. Raymond Wang, Metabolic/Lysosomal Disorders
Dr. Patti Dickson, Genetics/Lysosomal Disorders
Dr. Afshin Aminian, Orthopaedics
Dr. Agnes Chen, Neurology
Dr. Grace Mucci, Neuropsychology
Dr. Lynda Polgreen, Endocrinology
Dr. Michael Recto, Cardiology
Contact Us
An entire team of specialists from CHOC and Harbor-UCLA Pediatrics partner in one location to provide coordinated care for patients with MPS and other conditions that can result from the disease. We offer a unique expertise in MPS not seen at most hospitals.
To schedule an appointment at the MPS Multidisciplinary Center, please call 888-770-2462 and select “Specialty Care Clinics.” Please call 714-509-8852 with any additional questions about our program.