Spinal Muscular Atrophy (SMA)

ISIS-SMNRx to Be Tested in Phase 3 Trial in Infants With SMA

First Human SMA Gene Transfer Therapy Trial Opens

SMA: ISIS-SMNRx Shows Benefit in Infants, Children

New Forms of Genetic Testing Improve Diagnosis, Raise Questions

“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.

SMA: Olesoxime Results Look Promising

SMA: Interim Results Look Promising for SMN-Raising Drug

Interim results of a phase 2 clinical trial to test multiple doses of the experimental drug ISIS-SMNRx in infants with spinal muscular atrophy (SMA) suggest that the drug is well-tolerated and may prolong ventilator-free survival.

SMA: Need for SMN-Raising Treatment Highest Very Early in Life

Fairy of Hope


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 Imhoff

National Goodwill Ambassador

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.

Full name: 
Miss Reagan Imhoff
Reagan Imhoff
Spinal Muscular Atrophy (SMA)
Mixed Media

Blood-Filtering Procedure Removes Antibodies That Impede Gene Transfer

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.