Spinal Muscular Atrophy (SMA)

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.

Diamond in the Sunset

Artist: 

Amber attended MDA Summer Camp and painted this piece to donate to the MDA Art Collection. She began painting at age 8 and has received awards at local exhibits. Amber formed a team for the Eugene MDA Muscle Walk in February 2013 and prepared the "Dessert of the Month" for an MDA Fundraiser. Her favorite school subjects are history and science.

Amber Stock

2013
Full name: 
Amber Stock
Artist: 
Amber Stock
Disease: 
Spinal Muscular Atrophy (SMA)

Bikes

Sarah is attending Maryville University in Kirkwood, majoring in rehab services and political science. This photograph was taken with a Can PowerShot G12 camera. Sarah represented MDA as National Goodwill Ambassador in 2001. She previously donated “The Birthday Tree” to the MDA Art Collection which was featured in the 2001 MDA holiday card collection and the 2013 desk-top calendar.

Presents by The Tree

Sarah is attending Maryville University in Kirkwood, majoring in rehab services and political science. This photograph was taken with a Canon PowerShot G12 camera. Sarah represented MDA as National Goodwill Ambassador in 2001. She previously donated “The Birthday Tree” to the MDA Art Collection which was featured in the 2001 MDA holiday card collection and the 2013 desk-top calendar.

Mouse Model of Adult-Onset SMA Encourages Researchers

UPDATE: Pfizer and Repligen to Collaborate on SMA Therapy Development

The biotechnology firm Repligen today announced it has entered into an agreement with the global pharmaceutical company Pfizer to advance Repligen’s spinal muscular atrophy (SMA) program, which includes RG3039, an experimental drug for SMA whose development MDA has supported.

SMA: Full Speed Ahead

A Closer Look: SMA Slideshow

MDA Awards $8.5 Million to 31 Neuromuscular Disease Research Projects

In its summer 2013 round of research grant awards, the Muscular Dystrophy Association aims to catalyze research progress in a dozen neuromuscular diseases, with an eye toward applying that knowledge to related muscle diseases, as well.

“A large number of our grants are investigating new therapeutic technologies,” notes Jane Larkindale, MDA's vice president of research. “These are 'platform' technologies, where successes can be transferred well beyond the specific disease in which they are developed and tested.”

SMA — Bennett Novitch, Ph.D.

Bennett Novitch, assistant professor of neurobiology at the University of California, Los Angeles, was awarded an MDA research grant totaling $300,000 over a period of three years to study the development of motor neurons that control respiration and their significance for spinal muscular atrophy (SMA).

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