Your brave
little warrior.

Many times, children in the toughest situations display the most strength. And your little warrior is no exception. We know that a diagnosis of MLD can turn your world upside down. But we also know that your child’s strength is inspiring, and that every moment with him or her means the world to you. So as you consider whether to enroll your child in the Embolden Study – a clinical research study for MLD that may impact the way the condition is treated in the future – we want to share some information with you that will hopefully make your decision easier.

About the Embolden Study.

The Embolden Study is evaluating the safety and effects of a study drug on gross motor function (the abilities required to control the large muscles of the body for walking, running, sitting, crawling, and other activities) in children with late infantile MLD. This clinical research study has the potential to impact families in the future, and your child’s participation may contribute to bringing forth a treatment option for other children diagnosed with MLD. If enrolled, your child will receive the study drug and access to MLD-focused medical care at no cost to your family.

Who may qualify.

Infants and children who meet the following criteria, in addition to other criteria, may qualify for the Embolden Study:

  • Is male or female 18 months to six years old
  • Has been diagnosed with metachromatic leukodystrophy
  • Has family that is willing and able to comply with the study instructions

Study schedule.

Participation in the Embolden Study lasts approximately 116 weeks. During this time, you and your child will attend 110 study visits over four periods: the screening period, the baseline period, the treatment period, and the safety follow-up period.

Certain visits throughout the study will be completed at the main study site. Additional visits can be performed at a local site closer to your home. You will know which visits take place at the main site.