FA

News on Duchenne muscular dystrophy, Friedreich's ataxia, autoimmune diseases, and cell and gene therapies

posted on September 24, 2010 - 1:39pm
Duchenne muscular dystrophy
posted on January 1, 2011 - 4:54pm
QUEST Vol. 18, No. 1
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...
posted on January 1, 2011 - 4:51pm
QUEST Vol. 18, No. 1
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...
posted on January 1, 2011 - 4:46pm
QUEST Vol. 18, No. 1
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.

News on Duchenne muscular dystrophy, Friedreich's ataxia, autoimmune diseases, and cell and gene therapies

posted on September 24, 2010 - 1:39pm
Duchenne muscular dystrophy

News on amyotrophic lateral sclerosis, congenital muscular dystrophy, Friedreich's ataxia and spinal muscular atrophy

posted on August 27, 2010 - 11:20am
Amyotrophic lateral sclerosis (ALS)

Repligen Corp. is proceeding with development of an experimental compound to treat Friedreich's ataxia, with help from a new MDA grant.

posted on December 17, 2009 - 1:47pm
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

posted on September 29, 2009 - 11:32am
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