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Children with MPS IIIA suffer from severe and progressive neurocognitive decline, as well as multisystemic complications that can impact nearly all organ systems.1

Signs and symptoms
Learn about clinical presentation of MPS IIIA- Sanfilippo syndrome type A

Early diagnosis is critical for the management of MPS IIIA, yet symptom overlap with other neurocognitive disorders can delay diagnosis.3

Explore screening and diagnosis
Diagnosis journey of MPS IIIA- Sanfilippo syndrome type A

Comprehensive and individualized care is essential to MPS IIIA management,3 with a focus on optimizing function and maximizing quality of life for affected children and their families.3

Navigate MPS IIIA management
Navigate MPS IIIA - Sanfilippo syndrome type A disease management
Learn about clinical presentation of MPS IIIA- Sanfilippo syndrome type A

Children with MPS IIIA suffer from severe and progressive neurocognitive decline, as well as multisystemic complications that can impact nearly all organ systems.1

Diagnosis journey of MPS IIIA- Sanfilippo syndrome type A

Early diagnosis is critical for the management of MPS IIIA, yet symptom overlap with other neurocognitive disorders can delay diagnosis.3

Navigate MPS IIIA - Sanfilippo syndrome type A disease management

Comprehensive and individualized care is essential to MPS IIIA management,3 and should focus on optimizing function and maximizing quality of life for affected children and their families.3

Abbreviation: MPS IIIA, mucopolysaccharidosis type IIIA.

References

  1. 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/
  2. Lavery C, Hendriksz CJ, Jones SA. Mortality in patients with Sanfilippo syndrome. Orphanet J Rare Dis. 2017;12(1):168.
  3. Muschol N, Giugliani R, Jones SA, et al. Sanfilippo syndrome: consensus guidelines for clinical care. Orphanet J Rare Dis. 2022;17(1):391.