Spinal Muscular Atrophy (SMA)

SMA: Further Data on ISIS-SMNRx Shows Promising Results

Isis Pharmaceuticals, developer of the experimental spinal muscular atrophy (SMA) drug ISIS-SMNRx, revealed positive results from phase 2 clinical studies of the drug focusing on infants and children with SMA. The new data, which was shared on October 10 by ISIS representatives attending the 19th International World Muscle Society Congress in Berlin, included the following highlights:

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

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.

First Human SMA Gene Transfer Therapy Trial Opens

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.

SMA: ISIS-SMNRx Shows Benefit in Infants, Children

Update (Aug. 1, 2014): The phase 2 infant study of ISIS-SMNRx is now closed to new participants. However, a phase 3 study of this drug has opened. See ISIS-SMNRx to Be Tested in Phase 3 Trial in Infants With SMA.

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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

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.

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

Artist: 

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.

2014
Full name: 
Miss Reagan Imhoff
Artist: 
Reagan Imhoff
Disease: 
Spinal Muscular Atrophy (SMA)
Medium: 
Mixed Media

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