Wijburg FA, Aiach K, Chakrapani A, Eisengart JB, Giugliani R et al.

An observational, prospective, multicenter, natural history study of patients with mucopolysaccharidosis type IIIA.

Mol Genet Metab. 2022 ;135:133-142

Okur I, Ezgu F, Giugliani R, Muschol N, Koehn A et al..

Longitudinal Natural History of Pediatric Subjects Affected with Mucopolysaccharidosis IIIB.

J Pediatr. 2022 :S0022-3476(22)00538-8

Hoffmann F, Hoffmann S, Kunzmann K, Ries M.

Challenging behavior in mucopolysaccharidoses types I-III and day-to-day coping strategies: a cross sectional explorative study. Orphanet J Rare Dis. 2020 ;15:275

de Castro MJ, Del Toro M, Giugliani R, Couce ML.

Gene Therapy for Neuronopathic Mucopolysaccharidoses: State of the Art.

Int J Mol Sci. 2021 ;22:9200

Muschol N, Giugliani R, Jones SA, Muenzer J, Smith NJCet al;

MPS III Guideline Development Group. Sanfilippo syndrome: consensus guidelines for clinical care.

Orphanet J Rare Dis. 2022 ;17:391.