209 – How a Mom Launched the First Drug Approval in FA

March 13, 2023 twodisableddudes

In this Episode:

  • You Got This, Mental Health – Feeling seen and heard.  Some of the responsibilty is on you.
  • Can a single person make a difference? The amazing story of the first approved drug for Friedreich’s ataxia (FA)
  • Thank you notes – Reata Pharmaceuticals, and Kyle’s landlord at work.

As the story goes, a concerned mother, in an effort to do whatever she could for her son who has Friedreich’s ataxia (FA), connected a few important dots about science and the pharmaceutical industry.  When she decided to make a phone call to the Friedreich’s Ataxia Research Alliance (FARA), she started a series of actions and events that eventually led to the approval of the first treatment for FA, SKYCLARYS.  Listen for the details.

Links and Resources:

2 Comments on “209 – How a Mom Launched the First Drug Approval in FA

  1. Incredible interview and story guys! I never had a clue Ruth is the “Lorenzo’s Oil” parent of the 21st century. Kudo’s to her!

    And it warms my heart that she felt such support from FAPG in those early days.

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