Families fight SMA together.
Does your child havespinal muscular atrophy?
If your child is younger than 6 months of age and has been diagnosed with SMA Type 1, local doctors need your family’s help studying an investigational therapy for the treatment of spinal muscular atrophy.
Qualified participants in this research study will receive all study-related procedures, exams and medications at no cost. Travel assistance may be available for qualified families who don’t live near one of our research centers.