Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Genzyme, a Sanofi Company
Age
18–any
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
The patient or patient's legal guardian must provide signed, informed consent prior to performing any study-related procedures.
The patient must reside in the US.
The patient must have a confirmed diagnosis of Pompe disease defined as documented acid alpha-glucosidase (GAA) enzyme deficiency from any tissue source and/or GAA gene mutations.
The patient must have/had documented clinical signs and symptoms of Pompe disease.
The patient must have/had prior treatment with alglucosidase alfa produced at commercial scale OR be naive to enzyme replacement therapy (ERT) for the treatment of Pompe disease and meet at least 1 of the following criteria: require a wheelchair OR require some respiratory assistance for any number of hours (including night time) through non-invasive ventilation.
The patient must be capable of complying with the required program schedule of assessments.
You may not be if
Females who are pregnant or lactating
The patient has a clinical condition unrelated to Pompe disease that would interfere with program assessments.
The patient is currently enrolled in any clinical studies.
Clareo Health | Alglucosidase Alfa Temporary Access Program
