Login / Signup

Growth charts for patients with Sanfilippo syndrome (Mucopolysaccharidosis type III).

Nicole M MuscholDaniel PapeKai KossowKurt UllrichLaila Arash-KapsJulia B HennermannRalf StückerSandra Rafaela Breyer
Published in: Orphanet journal of rare diseases (2019)
MPS III is a not yet treatable severe neuro-degenerative disease, developing new therapeutic strategies might change the course of the disease significantly. The present charts contribute to the understanding of the natural history of MPS III. Specific growth charts represent an important tool for families and physicians as the expected height at physical maturity can be estimated and therapeutic effects can be monitored.
Keyphrases
  • type iii
  • primary care
  • body mass index
  • physical activity
  • early onset
  • replacement therapy