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Optimal care relies on a multidisciplinary approach1

Currently, MPS IIIA management is supportive and focused on treating symptoms and preventing complications.1 Comprehensive care should ideally be started immediately after diagnosis with the goal of maximizing peak abilities, slowing decline, and improving quality of life.1

Establishing a multidisciplinary care team is critical to managing the varied manifestations of the disease.1

Diverse team icon
MPS IIIA care involves a diverse team of specialists, which may include the following:1,2
  • Pediatrician
  • Pediatric neurologist
  • Otolaryngologist
  • Orthopedist
  • Physical therapist
  • Occupational therapist
  • Speech therapist
  • Audiologist
  • Dietitian
  • Psychologist
  • Social worker
  • Special educator

Disease monitoring and an individualized approach to care1

Given the progressive nature of MPS IIIA, continuous monitoring is necessary to determine the extent of disease progression and identify appropriate supportive interventions that meet the evolving needs of the child.1

Disease monitoring icon
Ongoing monitoring1,3
  • Physical, behavioral, developmental, and psychiatric assessments
  • Imaging studies
  • Laboratory work
Supportive interventions icon
Common supportive interventions1,3
  • Neurodevelopmental support (eg, speech, physical, and occupational therapy)
  • Medications to treat symptoms
  • Durable medical equipment (eg, wheelchairs, adaptive strollers, bath chairs)
  • Other support (eg, hearing aids, enteral feeding tube placement, ventilation tubes, surgical intervention for orthopedic complications)
Burden of care on families

Caring for a child with MPS IIIA places an enormous burden on families1,4

The ongoing psychologic stress and physical exhaustion experienced by caregivers can lead to feelings of isolation, depression, and post-traumatic stress disorder.1,4

Explore MPS IIIA resources

Abbreviations: MPS IIIA, mucopolysaccharidosis type IIIA.

References

  1. Muschol N, Giugliani R, Jones SA, et al. Sanfilippo syndrome: consensus guidelines for clinical care. Orphanet J Rare Dis. 2022;17(1):391.
  2. Cleveland Clinic. Sanfilippo syndrome. Reviewed July 24, 2024. Accessed November 26, 2024. https://my.clevelandclinic.org/health/diseases/sanfilippo-syndrome
  3. Wagner VF, Northrup H. Mucopolysaccharidosis type III. 2019. In: Adam MP, Feldman J, Mirzaa GM, et al, eds. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2024. Accessed January 2, 2025. https://www.ncbi.nlm.nih.gov/books/NBK546574/
  4. Shapiro E, Lourenço CM, Mungan NO, Muschol N, O’Neill C, Vijayaraghavan S. Analysis of the caregiver burden associated with Sanfilippo syndrome type B: panel recommendations based on qualitative and quantitative data. Orphanet J Rare Dis. 2019;14(1):168.