Families fight
SMA together

Does your child have spinal muscular atrophy?

If your child is younger than 5 years of age, local doctors need your family’s help studying an investigational therapy for the treatment of SMA in children who can sit independently but cannot stand or walk.

Qualified participants in the STRONG 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.

Find a Research Study Location

If you are interested in learning more about SMA research, the STRONG research study, and possibly enrolling your child in the study, enter your location in the field below to find the study site nearest to you. If travel costs are a concern, the study team can discuss options for you and your family.

Enter your location.

    View all STRONG research study locations

    ABOUT THE STUDY

    THE ONLY SMA GENE THERAPY IN HUMAN TRIALS

    AVXS-101, the therapy evaluated in this study for SMA patients, has been granted Orphan Drug Designation for the treatment of all types of spinal muscular atrophy (SMA) and Breakthrough Therapy Designation by the FDA.

    THE HEREDITARY NATURE OF SMA.

    Inheriting a mutated or missing SMN1 gene prevents the body from adequately producing the SMN (survival motor neuron) protein—which is critical to the function of nerves that control our muscles—leading to the debilitating muscle weakness caused by SMA.

    That’s why we need your family’s help with the STRONG research trial studying the effectiveness of gene therapy in the treatment of SMA. In this study, the gene therapy will be injected once near the lower end of the spinal cord in a procedure called an intrathecal injection; it will deliver a new gene that can produce the Survival Motor Neuron (SMN) protein that is missing in the spinal cord and motor nerves of children with SMA. By producing the SMN protein, the new gene may help improve a child’s SMA symptoms. 

    Eligible 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 sites.

    ABOUT RESEARCH STUDIES

    AveXis is a clinical-stage gene therapy company, dedicated to developing novel treatments and making them available to patients suffering from rare and life-threatening neurological genetic diseases.

    In order to provide new therapies to the public, pharmaceutical companies must test their investigational products very carefully through medical research studies (also called “clinical trials”). The United States Food and Drug Administration (FDA) thoroughly reviews the data collected in these studies before approving new medications for the public. Once the FDA approves a therapy, many pharmaceutical companies continue to conduct research on the approved product to collect ongoing safety and effectiveness data.

    Study doctors are required to follow strict rules to protect the safety of the people who volunteer to participate in clinical trials. All researchers must follow a detailed plan, called a protocol, which explains all study procedures and has been reviewed and approved by an independent board (or group of people knowledgeable about research) that oversees the safety of all study participants. Additionally, before enrolling individuals into a study, researchers must fully explain the study to the individual and answer any questions he or she may have. Participation in a clinical trial is voluntary, and individuals may withdraw from the study at any time and for any reason.

    For more information about AveXis, visit AveXis.com.

    For more information about this clinical trial, please visit www.clinicaltrials.gov.

    FAQS

    Pharmaceutical companies are required to conduct research studies to learn more about study treatments before they are approved and made available to the public. The results of this study will provide more information about the safety and effectiveness of the gene therapy. By taking part in this study, you and your child will make an important contribution to Spinal Muscular Atrophy (SMA) research.

    Currently there is one approved treatment available for SMA. Researchers are continuing to explore treatment options for SMA. You should discuss other potential treatments with your child’s doctor when considering this study.

    Although there is one other approved treatment option available, this treatment is the only gene therapy for SMA currently in development. The goal of this therapy is to deliver a new SMN1 gene that can produce the Survival Motor Neuron (SMN) protein that is missing in children with SMA. By producing the SMN protein, the new gene may help improve a child’s SMA symptoms.

    SMA is a severe neuromuscular disease caused by a genetic defect in the SMN1 gene—leading to the loss of motor neurons and resulting in progressive muscle weakness and atrophy. SMA is divided into sub-categories—SMA Types 1, 2, 3, and 4—based on age of disease onset, and SMN2 copy number, which generally correlate to survival motor neuron (SMN) protein levels.

    You can find a list of physicians participating in the study HERE. If there is not a study site located near you, you may be eligible for travel assistance. Please contact the study site closest to you for more information on travel assistance.

    If your child is eligible, and you agree to allow them to participate, he or she will be injected once near the lower end of the spinal cord in a procedure called an intrathecal injection to receive the gene replacement therapy. After treatment, your child will stay overnight at the hospital up through 48 hours for observation and evaluation. After your child is released from the hospital, you will be asked to bring them to follow-up visits for the remainder of the study.

    During study visits, doctors and study staff will also conduct various tests and assessments to evaluate your child’s health and progress. Some of these tests and assessments include, but are not limited to:

    • Review of your child’s medical history
    • Physical exam and vital signs (measurements of weight, length, blood pressure, heart rate, and temperature)
    • Spinal x-rays
    • Lung exams
    • Heart assessments
    • Blood and urine sample collections
    • Physical Therapy Assessments

    Total study participation will last about 12 months and include approximately 17 visits to the study clinic.

    Yes. Your child may receive the lower dose (Dose A) or the higher dose (Dose b) depending on timing of enrollment, and also age of your child at the time of enrollment.

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

    As with any research study, your child’s SMA may not improve as a result of study participation; all of the possible effects of the treatment are not known at this time.

    It is possible your child could experience a reaction to the gene replacement therapy, injection, or assessments. The study staff will provide you with a complete list of known risks related to this study.

    Because research studies can affect the health and safety of participants, your child will be closely monitored during this study. Researchers for this study designed a protocol, which explains all of the study procedures in complete detail. An independent review board responsible for participant safety reviewed and approved this protocol and requires that it be followed exactly.

    You may contact one of the study staff members at participating study sites for further information on this study. A list of study locations can be found HERE.

    ADVOCACY GROUPS

    Cure SMA, USA

    curesma.org

    SMA Foundation, USA

    smafoundation.org

    TREAT-NMD Neuromuscular Network, USA

    treat-nmd.eu

    Muscular Dystrophy Association (MDA), USA

    mda.org