Study type

Study type

Non-interventional study

Scope of the study

Effectiveness study (incl. comparative)
Safety study (incl. comparative)
Non-interventional study

Non-interventional study design

Other

Non-interventional study design, other

Observational long term follow up
Study drug and medical condition

Name of medicine

LIBMELDY

Medical condition to be studied

Metachromatic leukodystrophy
Population studied

Age groups

Infants and toddlers (28 days – 23 months)
Children (2 to < 12 years)
Adolescents (12 to < 18 years)
Adults (18 to < 46 years)

Estimated number of subjects

72
Study design details

Main study objective

Evaluate the durability of clinical efficacy and long-term safety following treatment with Libmeldy.

Data analysis plan

Full statistical methods are detailed in a separate statistical analysis plan (SAP).