Learn about currently enrolling spinal muscular atrophy (SMA) research studies

Welcome to Study SMA Now. Your child may be eligible for one of the SMA research studies presented below: SPR1NT, STR1VE-EU, or STRONG.

Click on one of the currently enrolling research studies to learn more.

SPR1NT SMA Research Study logo

GLOBAL

For babies who:

  • Are less than 6 weeks old
  • Have 2 to 4 copies of the SMN2 gene
STR1VE SMA Research Study logo

EUROPE ONLY

For babies who:

  • Are less than 6 months old
  • Have 1 or 2 copies of the SMN2 gene
STRONG SMA Research Study logo

USA ONLY

For children who:

  • Are less than 5 years of age
  • Have 3 copies of the SMN2 gene
  • Are able to sit independently but not stand or walk

ABOUT RESEARCH STUDIES

AveXis is a gene therapy company that is working to develop new treatments for patients with rare and life-threatening genetic diseases, like SMA. Research studies—also known as clinical trials—must be conducted to test experimental treatments before they can be used by the public.

The results of the SMA clinical trials will provide data about the safety and the effectiveness of gene replacement therapy in SMA. Participation in a research study is voluntary, and individuals may withdraw from the study at any time and for any reason.

The institutional review board or ethics committee protocol for research studies

Protecting study participants

Study doctors are required to follow rules while running research studies, including SMA research. These rules are meant to protect the safety of the people who volunteer to be in them. All researchers follow a detailed plan—called a protocol—which explains the steps of the study. This plan has been reviewed and approved by a group of people knowledgeable about research, called an institutional review board (IRB) or ethics committee (EC). The IRB and EC are concerned with protecting the welfare, rights, and privacy of study participants. Before you volunteer to join a research study, the researchers must fully explain the study and answer any and all questions that come up.

By taking part in a research study, you and your baby will play an important role in the future of SMA and potential new treatments.

Back to SMA studies

Resources

Cure SMA

Cure SMA is dedicated to the treatment and cure of SMA. They fund research and provide families with support.

curesma.org

SMA Foundation

The mission of the SMA Foundation is to accelerate the development of a treatment for SMA.

smafoundation.org

TREAT-NMD Neuromuscular Network

TREAT-NMD is a network for the neuromuscular field that provides a system to ensure that the most promising new therapies reach patients as quickly as possible.

treat-nmd.eu

Muscular Dystrophy Association (MDA)

MDA is leading the fight to free individuals—and the families who love them—from the harm of muscular dystrophy, amyotrophic lateral sclerosis, and related muscle-debilitating diseases that take away physical strength, independence, and life. It works to find research breakthroughs across diseases, provides support for individuals from day one at MDA Care Centers, and empowers families with services and support in hometowns across America.

mda.org