Spinal Muscular Atrophy (SMA)

MDA Funds Development of Utrophin 'Magnet' for DMD/BMD


Phase 1 Trial Opens of ISIS-SMNRx in Children with SMA

A 24-person, phase 1 trial to test the safety and tolerability of the experimental drug ISIS-SMNRx in children with spinal muscular atrophy (SMA) is now open at Columbia University Medical Center in New York, with additional sites expected to open in Boston, Philadelphia, Dallas and Salt Lake City.

PTC, Roche Team Up Against SMA

Swiss biotechnology company Roche, and PTC Therapeutics in South Plainfield, N.J., on Nov. 29, 2011, announced a collaboration through which the two companies will work together to advance drug development for spinal muscular atrophy(SMA).

Systemic Antisense Injections Rescue Severe SMA Mice

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.

Research Briefs: Spinal Muscular Atrophy

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.

Induced Stem Cells Require Cautious Approach

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

Tabi Estrellado: Writing Code, Writing Songs, Winning Awards

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.

Trainer with SMA Turns ‘Underdogs’ into Winners

From the moment Linzey Zoccola received her first service dog at age 16, she knew what she wanted to do with her life.

Zoccola has type 2 spinal muscular atrophy and has used a power chair since the age of 4. She has always been strongly independent, but having a service dog was a revelation.

Here was a creature that not only brought her joy and companionship, but also helped her perform tasks at home and school, achieve greater independence, and even smoothed her path socially.

Spinal Muscular Atrophy Care Guidelines

The Family Guide to the Consensus Statement for Standard of Care in Spinal Muscular Atrophy was prepared by SMA Advocates for families affected by SMA. The full, 22-page text of the Consensus Statement was published in the August 2007 issue of the Journal of Child Neurology. Both are available as PDFs by following the links above.

Last Updated: 
Sat, 11/03/2007 - 14:34