Three years ago, when our daughter Annie was nine, our lives changed forever as she was diagnosed
with an unusually rare disease called Friedreich’s Ataxia.
Friedreich’s Ataxia (FA) is a degenerative neuromuscular disease. Its effects are devastating.
Initially impacting balance and coordination, the disease then leads to long-term medical
conditions; scoliosis, diabetes, vision and hearing loss, as well as serious heart conditions. Like
Annie, patients are generally diagnosed between the ages of 5 and 15. While FA progresses at a
different pace for each individual, most require a wheelchair ten years after diagnosis and face an
extremely shortened life expectancy. Annie is one of 15,000 people living with this debilitating
As we tried to digest and make sense of this new reality, our family remained very private honoring
Annie’s request that we tell no one she is any different from her friends. As much as we want to
continue to respect her wishes, we feel the time has come to share this diagnosis and to ask for
your support now and not five years from now when the disease may have progressed too far.
Since receiving Annie’s diagnosis we have dedicated our lives to finding a cure - spending most
waking (and many sleeping) moments reading, meeting and strategizing ways to treat and to combat
FA. Tom immediately joined the executive board of the Friedreich’s Ataxia Research Alliance (FARA),
on which he still serves. In February 2014, together with Karen’s parents and FARA, we opened
the Center of Excellence at the Children’s Hospital of Philadelphia/University of Pennsylvania
where doctors and scientists work exclusively on FA. And last fall, Tom launched a biotech company
that is moving a treatment into clinical trials.
Most recently we established The CureFA Foundation to advance and support FA research. We are
fortunate that, unlike most other debilitating diseases, FA is a single gene defect and that gene
has been identified. Researchers rarely use the word “cure” when studying a rare disease with no
approved therapies, but with FA that word is now a part of the lexicon. With the many clinical
trials and cutting edge therapies currently in place, we feel the time is now to make a significant
investment in advancing FA research. At this critical juncture, we know we can change the course of
Friedreich’s Ataxia, which is why we are asking for your support now. A cure is within our reach.
We know that no life is without challenge and that difficult times can strengthen and can bring
unexpected grace. This diagnosis has given us the gift of perspective, allowing us to see and to
value what is most important. We have been blessed with four wonderful children, a strong faith, a
loving family, and truly supportive friends. Although Annie can no longer play lacrosse, tennis or
dance as she once did, she is happy. Her strong sense of self, buoyant spirit, and ‘stubbornness’
are serving her well. Her friends and those she loves are everything to her. They are everything to
On behalf of Annie and the many thousands of children and families living with FA, we extend our
deepest thanks for your support.
Karen and Tom Hamilton