Pharmaceutical Research and Manufacturers of America (PhRMA) President and CEO Billy Tauzin today honored Ron and Raychel Bartek for their dedicated work driving awareness of Friedreich's ataxia, a debilitating, life-threatening, rare disease.
The Barteks are co-founders of the Friedreich's Ataxia Research Alliance, an organization dedicated to the pursuit of scientific research leading to treatments and a cure for the disease. However, they are more than advocates; they are also parents of Keith Bartek, who succumbed to Friedreich's ataxia recently at the age of 24.
Although there remains no approved medication to treat the disease, clinical studies are currently being conducted. However, such near-term hope was not always the case, as Raychel Bartek said: "When Keith was diagnosed, I asked the doctor what medicine she would prescribe for him. Her eyes were watering when she said, 'There's nothing he can take.'"
Regarding the lack of treatment options, Ron Bartek added, "The prognosis was awful for Keith."
Unfortunately, millions of families have experienced the same frightening diagnosis of a rare disease that doesn't have any known treatments. In fact, there are more than 1,200 recognized rare diseases, defined as those that affect less than 200,000 people in the U.S. According to the National Organization for Rare Diseases (NORD), roughly 25 million Americans suffer from such a condition.
Like his parents, Keith dedicated his life to raising awareness of the disease, hoping to drive research. "He may have only lived 24 years, but in those 24 years he inspired a lot of people to do the right thing and to accelerate research," Raychel Bartek explained. "Twenty-four years isn't a lot of time for any of us, but he had a purpose in his life and he knew that when he died."
"Because of the relatively small number of patients affected by these rare diseases, many have been historically under-treated," said PhRMA's Tauzin. "That's why America's pharmaceutical research and biotechnology companies continue their commitment to patients suffering from these so-called 'orphan diseases,' and much of that commitment has been facilitated by the Orphan Drug Act."