Diagnosed as bipolar and autistic while still a child, Bridget Morris grew up with a host of developmental problems. That’s why, when she was tested for Friedreich’s ataxia (FA) at age 10, her mother, a nurse, was “very cavalier.”
Bridget had muscle tightness, weakness and scoliosis (a curved spine), her handwriting was worsening, and she had begun to fall and walk with a “wide” gait to steady...
Pinalben “Pinky” Patel, 28, was born in a village in Western India and moved to the United States with her family four years later. She already was starting to show symptoms of Friedreich’s ataxia (FA).
“My aunt and cousin took me trick-or-treating for the first time,” she says. “When we got home, my aunt complained that I kept falling down on purpose for attention. A couple years later my...
When Kayla Prather was 8 years old, she began having trouble with her balance. Her mom took her to a pediatrician, who tested her reflexes and found she didn’t have any.
More tests followed, including one of Kayla’s DNA, and the diagnosis of Friedreich’s ataxia (FA) was confirmed.
Overall, idebenone has been shown to be safe and well-tolerated in a number of phase 1, 2 and 3 human clinical trials dating back to 1999. The most common side effects are mild to moderate gastrointestinal issues such as diarrhea, nausea and indigestion. Early trials at low doses first demonstrated the potential for idebenone to reduce FA-associated cardiomyopathy (heart muscle abnormality).
Repligen Corp. is proceeding with development of an experimental compound to treat Friedreich's ataxia, with help from a new MDA grant.
Development of a promising experimental medication to treat Friedreich's ataxia (FA) is proceeding, with help from a $731,534 grant MDA awarded to Repligen Corp. of Waltham, Mass., this month (December 2009).
This is the second research grant that MDA has awarded to the small biopharmaceutical company, through the Association's translational (laboratory-to-clinic) research program.
A compound being developed to treat Friedreich's ataxia specifically targets an unwanted molecular brake
Scientists at the Scripps Research Institute in La Jolla, Calif., and the Repligen Corporation in Waltham, Mass., have identified the precise biochemical brake that limits production of a needed protein in Friedreich's ataxia (FA) and determined that this brake is specifically targeted by an experim