The calendar features entire weeks, or even months, that draw attention to diseases such as diabetes, cancer and heart disease.
But comparatively little notice is paid to juvenile Batten disease, a rare, fatal, inherited neurological disorder.
That may be about to change.
“Families of children with juvenile Batten disease are working tirelessly to let everyone know how devastating this disease can be and to help raise money to find a cure,” says Mary Beth Kiser, president and CEO of the Beyond Batten Disease Foundation (www.beyondbatten.org), a non-profit that is one of the leaders in that effort.
“Most Americans probably never heard of juvenile Batten disease. But for the families who face it, it is devastating.”
Children with the incurable disease usually start to show symptoms between ages 5 and 10. They suffer progressive vision loss, commonly followed within a couple of years by seizures and, as time passes, the loss of motor functions and speech. Psychosis can appear at any time in the disease. The children can expect to die prematurely, usually in their late teens or early 20s.
Juvenile Batten is the disease that that was responsible for the July deaths of three siblings over the course of three days in Utah.
Here are just a few facts about juvenile Batten:
There is more than one form of Batten. Juvenile Batten is just one form of the disease. Two other classic forms are infantile and late infantile. Infantile Batten shows up between 12 months. Children die around age 4. Children with late infantile Batten begin showing symptoms about age 4. They typically die when they are about 8 to 10 years old. Adult Batten shows up when people are 25 to 43 years old. Those with adult Batten have a normal life span. The various forms of the disease occur in about two to four out of every 100,000 live births in the United States.
How it is treated. No specific treatment is known that can halt or reverse the symptoms of any form of Batten disease. However, seizures can sometimes be reduced or controlled with anticonvulsant drugs, and psychiatric and motor problems can be managed with medication as they arise. Physical therapy and occupational therapy may help patients retain motor functioning as long as possible.
Research and the quest for a cure. Medical researchers are hard at work seeking a cure. In the meantime, progress is being made on research into a treatment that would slow the progress of the disease, buying time for juvenile Batten patients until that cure is found. Kiser says the foundation is involved in a massive fund-raising effort to help hurry along the research for that treatment by getting it through an expensive validation and development process so that it can receive FDA approval for clinical trials.
“The families of children with juvenile Batten are determined to fight back against this disease and to bring awareness to the effort to find a cure,” Kiser says. “But for each of these families, it’s a battle with a deadline. They don’t have much time.”
Mary Beth Kiser, the president and CEO of the Beyond Batten Disease Foundation (www.beyondbatten.org), leads the foundation’s efforts to raise awareness and funds for research for a cure for juvenile Batten disease.