California-based Isis Pharmaceuticals has announced the opening of a phase 3 clinical trial of its experimental drug ISIS-SMNRx in infants with spinal muscular atrophy (SMA), a muscle-weakening disease that results from loss of nerve cells in the spinal cord.
A phase 1 trial to test the safety and efficacy of gene transfer therapy in infants with type 1 spinal muscular atrophy (SMA) who are 9 months old or younger has opened at Nationwide Children's Hospital in Columbus, Ohio, under the direction of neurologist Jerry Mendell.
“Knowing, if not all, is almost all,” said Matthew Harms, a neurologist and neurophysiologist from Washington University in St. Louis, in his presentation on genetic testing for neuromuscular disorders at the 2014 MDA Clinical Conference, held in Chicago March 16-19.
Update (May 2, 2014): Trophos, developer of olesoxime to treat SMA, announced more detailed results from this trial at the spring 2014 annual meeting of the American Academy of Neurology and in an April 28, 2014, press release.
Reagan is MDA’s 2014 National Goodwill Ambassador. Prior to that she served as the Wisconsin State Goodwill Ambassador for three years. Her favorite subjects are music, art and reading. She began drawing when very young. Reagan’s other “favorite” activity is dancing. She appeared on the 2012 MDA Show of Strength and performed a dance from her wheelchair dressed in a fairy costume.
In this artwork, Reagan drew her friends in the following order: Bryson, Abbey, Ryder and herself.
Reagan is a charming, happy and outgoing third-grader who enjoys art, social studies and recess. She takes dance lessons, attends art class and loves spending time with her family and friends. Reagan dreams of being an artist, a dancer and a nurse.
A research group supported in part by MDA has found that a blood-filtering strategy known as plasmapheresis looks promising as a way to help overcome one type of unwanted immune response to gene transfer therapy. Gene transfer therapy is the addition of therapeutic genes to treat disease, and it is often administered via transport vehicles made from viruses.
Louis Chicoine at Nationwide Children's Hospital and Ohio State University in Columbus, Ohio, and colleagues, reported the findings online Oct. 23, 2013, in the journal Molecular Therapy.