Our Story
Three years ago, a family's life changed forever when their daughter, Annie, was diagnosed with Friedreich’s Ataxia (FA), a rare degenerative neuromuscular disease. This condition affects balance and coordination, leading to severe long-term medical issues such as scoliosis, diabetes, and heart conditions. With a diagnosis typically occurring between ages 5 and 15, most patients require a wheelchair within ten years and face a significantly shortened life expectancy. Annie is one of 15,000 individuals worldwide living with this debilitating condition.
Initially, the family chose to keep Annie's diagnosis private, respecting her wishes to be seen as no different from her friends. However, they realized the urgency of sharing her story and seeking support to combat FA before it progresses too far. Since Annie's diagnosis, they have dedicated their lives to finding a cure, engaging with the Friedreich’s Ataxia Research Alliance (FARA) and opening the Center of Excellence at the Children’s Hospital of Philadelphia/University of Pennsylvania, where dedicated professionals focus on FA research.
In 2014, the family launched a biotech company to advance treatment options, and most recently, they established the CureFA Foundation to further support FA research. Unlike many other diseases, FA is caused by a single gene defect, which has been identified, making the prospect of a cure more tangible. With ongoing clinical trials and innovative therapies, the time to invest in FA research is now.