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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

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