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Participation in Clinical Trials

The objective of these educational materials is to prepare you for what could be one of the biggest decisions of your, or your child’s, life - whether to enroll in a clinical study of an investigational drug or gene therapy. Please click on the icons below to learn more information.

We hope you find resources here to help you make the best decision for yourself or for your child. Please email if you have questions.

The terminology and abbreviations associated with FA clinical management and research can be unfamiliar. You can find a glossary HERE.

Video resources are available explaining the genetics and inheritance of Friedreich’s ataxia. These videos can be viewed at; click on the tab “Effect of genetic mutations”.

The list on the right-side of this page includes the active & enrolling clinical studies.

Basics of Drug Development
FAQ for Clinical Trial Participation

Important Message on Clinical Trial Participation

Overview of
Gene Therapy for FA

The lessons below were developed in collaboration with Salem Oaks. Each lesson will open in a new tab in the Salem Oaks website.
We recommend that you view the lessons in sequence. The information builds on each lesson and subsequent lessons refer to information in previous lessons. You will need to set up an account in the Salem Oaks website when you view the first lesson. There is no charge to engage in the gene therapy education. Please send any questions to

Lesson 1: Foundations of Drug & Gene Therapy Clinical Trials
Sets the stage with some fundamentals about small molecule drugs and gene therapy approaches to treating Friedreich’s ataxia.
Lesson 2: Benefit-Risk Assessments
Compares the general risks and benefits of drug and gene therapy clinical studies.
Lesson 3: Bringing the Gene Therapy Decision to Life (a short video)
A video of parents receiving the unexpected diagnose of Duchenne Muscular Dystrophy for their son and being presented with the option of enrolling him in a gene therapy clinical trial.
Lesson 4: Understanding Informed Consent
Presents the key concepts of the informed consent process for a gene therapy clinical trial.
Lesson 5: Making Informed Decisions
Presents tools for how to work through your decision-making to arrive at the course of action that is best for you or for your child regarding gene therapy clinical trial participation.

Appreciation of the Drug Development Process through Understanding Clinical Trials

To learn more about Drug Development and participation in Clinical Trials - Click Here.
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