We are seeking the views of parents of children with Friedreich’s ataxia (and children themselves) on behalf of a pharmaceutical company who have a programme in Friedreich’s ataxia.

The question relates to what would be the ideal formulation for future medication. Any help you can give in designing medication for children with FA by filling out the below questions would be gratefully received.

Question 1: What would be the ideal formulation of medication for children with Friedreich’s ataxia?

Please indicate the preference rating the following in order of preference (1 being the most preferable and 5 the least)

  • Liquid (must refrigerate)
  • Liquid (non-refrigerate)
  • Capsules (can sprinkle on palatable food or mix with drinks, such as juice)
  • Pouch/sachet (can sprinkle or mix)
  • Chewable tablets

 

Question 2: What is the age of your child with Friedreich’s ataxia?

If you have additional comments please let us know.

Please send responses to Julie Greenfield, Head of Research at Ataxia UK ([email protected]).

Thank you in advance for your willingness to provide advice.