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FA?

Friedreich's Ataxia (FA) is a rare genetic disease that damages the nervous system, causing progressive loss of coordination and movement. Loss of ambulation occurs 6 years earlier in patients with symptom onset before the age of 15.1* That's why PTC Therapeutics has worked closely with the FA community for over a decade to better understand their challenges, particularly in managing younger, more vulnerable patients. Now we need your help. Take our short survey.

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Share your thoughts

Please take a moment to share your insights and experiences with managing FA in pediatric patients to help us better understand the challenges and opportunities young patients with FA face.

1What do you see as the most important unmet need in managing FA that you would like to see addressed?

2Which of the following age range of pediatric FA patients do you see most often in your practice?

3For patients 16 years and older diagnosed with FA, do you treat with prescription medication?

4Which of the following is the top reason a patient might not start prescription treatment upon diagnosis of FA?

5Are you familiar with the PTC Phase 3 clinical trial in FA?

*Compared to patients 15−24 years of age in a Friedreich's Ataxia Clinical Outcome Measures Study (FA-COMS), a natural history study designed to assess progressive features leading to loss of ambulation in 1021 patients with FA over 4606 yearly follow-up visits.1

Reference: 1. Rummey C, Farmer JM, Lynch DR. Predictors of loss of ambulation in Friedreich's ataxia. EClinicalMedicine. 2020;8(18):1−9.