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 European Medicines Agency thoroughly reviews the data collected in these studies before approving new medications for the public. Once the EMA 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.