Study type

Study type

Non-interventional study

Scope of the study

Drug utilisation
Non-interventional study

Non-interventional study design

Cohort
Study drug and medical condition

Medical condition to be studied

Fabry's disease
Population studied

Age groups

Adults (18 to < 46 years)
Adults (46 to < 65 years)
Adults (65 to < 75 years)
Adults (75 to < 85 years)
Adults (85 years and over)

Estimated number of subjects

100
Study design details

Main study objective

Describe & estimate the frequency of pregnancy complications in women with Fabry disease who were exposed to at least 1 dose of migalastat during pregnancy & fetal/infant outcomes in infants through 1 year of age > pregnancy outcomes > fetal/neonatal and infant outcomes

Outcomes

Describe and estimate the frequency of pregnancy complications in women with Fabry disease who were exposed to at least 1 dose of migalastat during pregnancy and fetal and infant outcomes in infants through 1 year of age > pregnancy outcomes > fetal/neonatal and infant outcomes

Data analysis plan

A formal Statistical Analysis Plan (SAP) will include details of all planned analyses and presentation of study data. Since this is an observational study, descriptive analyses will be provided. Descriptive statistics will comprise the number of observations (n), mean, standard deviation (SD), median, minimum, and maximum for continuous variables, and n and percent for categorical variables.