Werdnig-Hoffmann Disease (Spinal Muscular Atrophy)

Phase 3 Trial of ISIS-SMNRx Now Open for Children with SMA

Update (Dec. 1, 2014): Isis has announced that its partner, Biogen Idec, plans to conduct a phase 2 trial of ISIS-SMNRx in up to 25 presymptomatic newborns genetically predisposed to develop SMA, as well as a phase 2 study of this drug in approximately 20 patients with infantile or childhood-onset SMA who do not meet the inclusion criteria for the current phase 3 studies. These two additional studies could begin in the first half of 2015.

PTC To Test RG7800 in SMA

PTC Therapeutics, a South Plainfield, N.J., biopharmaceutical company, says it will soon open a trial to test the safety and tolerability of its investigational drug RG7800 in adults and children with spinal muscular atrophy (SMA). The announcement was made in a Nov. 19, 2014, press release.

SMA Gene Associated with Sporadic ALS

Duplications (extra copies) of the SMN1 gene are a "major" risk factor for developing sporadic (noninherited) ALS (amyotrophic lateral sclerosis), a team of scientists based in the Netherlands and United Kingdom has reported.

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.

Living With

Dear Friends:

I’ve had symptoms of spinal muscular atrophy since I was a baby. When most children were beginning to walk, my parents noticed that my head would flop to the floor as I crawled.

Jerry Ferro

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Spinal Muscular Atrophy

Description: 

MDA leads the search for treatments and therapies for spinal muscular atrophy (SMA). The Association also provides comprehensive supports and expert clinical care for those living with SMA.

In this section, you’ll find up-to-date information about spinal muscular atrophy, as well as many helpful resources. This information has been compiled with input from researchers, physicians and people affected by the disease.