For babies who:
- Are less than 6 weeks old
- Have 2 to 4 copies of the SMN2 gene
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.
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.