Before starting the treatment, your child must not have received enzyme replacement therapy (medications called Replagal or Fabrazyme) for at least 14 days.

The medical team will confirm that your child has a specific gene variant that can respond to migalastat treatment.

Your child will undergo a complete baseline evaluation before starting the medication.

This will include blood tests to measure medication levels in the blood and check overall health.

Your child will have vital signs measured, including blood pressure, heart rate, and temperature.

A physical examination will be performed to assess overall health.

Your child’s weight and height will be measured and recorded.

An electrocardiogram will be performed to check the electrical activity of the heart.

An echocardiogram will be performed to examine the structure and function of the heart using ultrasound.

For girls aged 8 years and older and boys aged 9 years and older, a Tanner stage assessment will be performed to evaluate physical development.

Your child will receive migalastat hydrochloride in the form of a dispersible tablet that can be dissolved in liquid.

The medication is taken by mouth.

The specific dose will be determined based on your child’s age and weight to achieve the appropriate medication levels in the blood.

The treatment will continue for 12 months.

Throughout the 12-month treatment period, your child will attend regular monitoring visits.

At these visits, blood samples will be taken to measure pharmacokinetics, which means how the medication moves through and is processed by the body.

The medical team will monitor for any side effects or unwanted reactions to the medication.

Blood tests will be repeated regularly to check for changes in laboratory values.

Vital signs will be measured at each visit to track any changes over time.

Physical examinations will be performed regularly to assess your child’s overall health.

Weight and height will be measured at each visit to monitor growth.

Electrocardiograms will be performed periodically to monitor heart function.

For girls aged 8 years and older and boys aged 9 years and older, Tanner stage assessments will be repeated to track physical development.

The medical team will continuously monitor for any treatment-emergent adverse events, which are any health problems that occur during treatment.

Any serious adverse events, which are significant health problems requiring immediate attention, will be carefully tracked.

If your child experiences side effects that make it necessary to stop the medication, this will be documented.

At the end of the 12-month treatment period, your child will undergo a comprehensive final assessment.

This will include all the same tests performed at baseline: blood tests, vital signs, physical examination, weight and height measurements, electrocardiogram, and echocardiogram.

For girls aged 8 years and older and boys aged 9 years and older, a final Tanner stage assessment will be performed.

The medical team will compare all results from the final assessment with the baseline measurements to evaluate changes over the 12-month treatment period.

If your child is of reproductive age, a medically accepted method of contraception must be used throughout the entire study period.

Contraception must continue for up to 30 days after the last dose of migalastat.