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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 list on the right column of this page (at bottom on a narrow device) includes the active & enrolling clinical studies.

Clinical Trial Educational Materials

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Gene Therapy Educational Materials

Start Here Overview Video: 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.

Basics of Drug Development

You, your child or someone else you love has Friedreich's ataxia. Maybe you found out a year ago, maybe five, maybe you found out yesterday. Most likely by now you've read everything you could possibly read on the subject.

Or, possibly, you are still frantically researching. Despite the significant progress that has been made on the road to finding answers and solutions for FA, there still is no silver bullet, no “get out of jail free” card. And this is an incredibly vibrant and passionate research community, committed to finding treatments and ultimately a cure. So what's the hold-up? Why is it taking so long?

For any disease, whether cancer, muscular dystrophy or Friedreich's ataxia, there are phases of research that scientists must go through to understand a disease and discover potential treatments, as well as a series of steps before a drug or treatment can be prescribed to a patient. In this journey, the number one priority is to ensure patient safety. To be successful, each phase or step demands significant resources—financial, human and organizational. And the highest levels of scrutiny are required in order to ensure a safe and effective outcome. These activities must be done carefully, methodically, usually with several being done at the same time as scientists explore multiple treatment avenues. They explore drugs to slow or stop the underlying disease as well as drugs or interventions to reverse the disease. Additionally, a disease such as Friedreich's ataxia could benefit from the use of different drugs, each addressing specific aspects of the disease. If different drugs are used together, you might hear this referred to as the “cocktail” approach or the combined therapy approach.

Section contributed by Kristi Wright in collaboration with FARA. Kristi is the author of The Basker Twins in the 31st Century. 75% of all royalties from this novel are donated to FA research.

+ What We Understand
+ Developing the Research Questions
+ Drug Discovery & Development
+ Safety Testing
+ Drug Formulation & Quality Control
+ Putting Together a Clinical Trials Team
+ The Clinical Trial
+ Importance of Good Trial Design
+ Measuring the Results
+ Key Partnerships

Anyone considering participating in a clinical trial should discuss the matter with his or her physician.  FARA does not endorse or recommend any particular studies.

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