Natural course in Dutch patients with Sanfilippo syndome (MPS III)

Natural course in Dutch patients with Sanfilippo syndome (MPS III) - Natural course Sanfilippo

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

NL-OMON

Registry ID

NL-OMON31897

Enrollment

163

Registered

2008-05-27

Start date

2008-05-01

Completion date

Unknown

Last updated

2024-05-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

MPS III Mucopolysaccharidosis type III

Interventions

None listed

Eligibility

Age

2 Years to 99 Years

Inclusion criteria

Inclusion criteria: The patient should have a biochemically confirmed deficiency of heparan-N-sulfatase (MPSIIIA) or A-N-acetylglucosaminidase (MPS IIIB).

Exclusion criteria

Exclusion criteria: The parent/legal representative is unwilling to participate or no clinical information is available

Design outcomes

Primary

Measure	Time frame
Outcome of the study will be an increased knowledge on the natural course of Sanfilippo syndrome. More knowledge on symptoms and life expectancy. In addition knowledge on the pattern of GAG excretion in the urine will increase.	—

Countries

Netherlands

Outcome results