Treatment of a mouse model of severe spinal muscular atrophy (SMA) with an antisense oligonucleotide results in greater and longer-lasting benefit when given systemically than when given only to the central nervous system, new research shows.

When Vance Taylor was a boy, he didn’t know any adult he could look to and say, “There’s somebody like me.”

His mother, Morena Noyes, recalls the first time she took Vance and his sister Kathy — both of whom have limb-girdle muscular dystrophy— to MDA summer camp.

“We were still in the parking lot, in our Astro van,” Noyes says. “Vance looked, and then he turned to his sister and said, ‘Kathy, there are people like you and me here!’ He was just beaming.”

Many years ago, when the physician said that my son Joe, who had type 1 spinal muscular atrophy, perhaps would live until 2 years old, I went into “Rambo mode,” ready to attack anything that got close enough to harm him.

But as the years went by and Joe’s health held stable, I silently vowed I would do my best to allow him to live outside the sterile little bubble I had created around him.

Skeletal muscle damage reversed in SMA mice

In addition to muscle damage caused by the loss of motor neurons in the spinal cord, skeletal muscle degeneration inspinal muscular atrophy (SMA) also stems from low levels of the protein SMN in muscle fibers. Now a team of scientists from Germany and the United Kingdom reports that muscle damage caused by low SMN can be reversed with drugs.

Stem cells have been much in the news lately, including for neuromuscular diseases.

Tabitha Estrellado is living her dream. She’s a 27-year-old career woman with a nice apartment in Manhattan. She loves her job, has a sweet commute, and writes music and performs locally in her spare time.

The Muscular Dystrophy Association has awarded 40 research grants totaling $13.7 million to advance the understanding of disease processes and uncover new strategies for treatments and cures of muscular dystrophy and the more than 40 other diseases in the Association's program.

The new grants were recommended by MDA's Scientific and Medical Advisory Committees and approved by MDA's Board of Directors at its July 2011 meeting.