Log in

Is My Child Eligible?

To qualify for this study, your child must:

  • Be 2 to 12 years of age at screening
  • Be medically diagnosed with spinal muscular atrophy (SMA)
  • Have had onset of clinical signs and symptoms consistent with SMA at >6 months of age
  • Be able to sit independently, but has never had the ability to walk independently
  • Not have a medical need for ventilation for >6 hours during a 24 hour period, at screening
  • Not have a medical need for a gastric feeding tube, where most feeds are given this way
  • Not have severe contractures or severe scoliosis by X-ray examination at screening
  • Not have been hospitalized for surgery (including scoliosis surgery), pulmonary event, or nutritional support within 2 months of screening or planned during the length of the study
  • Not have an untreated or inadequately treated active infection requiring systemic antiviral or antimicrobial therapy at any time during the screening period
  • Not have a history of brain or spinal cord disease that would interfere with lumbar puncture procedures, CSF circulation, or safety assessments necessary for the study
  • Not have an implanted shunt for the drainage of CSF or an implanted CNS catheter
  • Not have a history of bacterial meningitis
  • Not have received this investigational medication in any previous clinical study
  • Not have had a prior injury (e.g., upper or lower limb fracture) or surgical procedure which affects your child’s ability to perform any of the outcome measure testing required in the protocol
  • Not have been treated with another investigational drug, biological agent, or device within 1 month of screening
  • Not have been treated with valproate or hydroxyurea within 3 months of screening
  • Not have had gene therapy, antisense oligonucleotide therapy, or cell transplantation
  • Be able to follow all study procedures
  • Meet additional study-related criteria

All study-related visits, tests, and medications will be provided to participants at no cost. In addition, reimbursement for study-related travel and expenses will be provided.

If you believe your child is eligible for this study, please click here to find a study clinic located near you.