Spinal Muscular Atrophy (SMA)

SMA: RG3039 Proves Safe in Phase 1 Trial

Update (June 21, 2012):This story was updated to include the fact that MDA funding helped support the phase 1 trial, as well as the preclinical development of RG3039.

Neuromuscular Disease Research Discussed at 2012 AAN Meeting

Top scientists and clinicians from around the world are discussing the latest research in neuroscience, and the care of individuals with nerve and muscle diseases, at the 2012 annual meeting of the American Academy of Neurology in New Orleans, April 21-28.

MDA 2012 Conference Report: Targeted Therapies

The progress of several experimental therapies currently in development for neuromuscular diseases was discussed at MDA's 2012 Clinical Conference, held in Las Vegas March 4-7.

SMA Disease Course May Be Affected by ZPR1

Adequate levels of zinc finger protein 1 (ZPR1) appear to be a "protective" modifier of spinal muscular atrophy (SMA), an MDA-supported team of scientists has reported.

Modifiers influence disease onset and severity by changing various biological pathways.

The identification of ZPR1 as a modifier reveals a potential target for therapy development and also sheds light on the mechanisms that drive SMA.

MDA 2012 Conference Report: Genetics and Immunology Update

More than 500 physicians, allied health care professionals and MDA staff attended the MDA's 2012 Clinical Conference in Las Vegas, March 4-7.

The program emphasized:

SMA Research Briefs: Antisense and Fasudil

Below are highlights of two recent studies in spinal muscular atrophy (SMA), a disease in which the nerve cells that control muscles (motor neurons) in the spinal cord die, causing progressive weakness in the voluntary muscles.

Under the Sea

Destiny has been drawing since age 3.  She has served as a local Goodwill Ambassador for several years.  Her hobbies include fishing, reading and swimming.

Destiny Sanchez

Location

Pineville, LA
2012
Full name: 
Destiny Sanchez
Artist: 
Destiny Sanchez
Disease: 
Spinal Muscular Atrophy (SMA)
Medium: 
Acrylic

Sunset at Camp Marie

Mathew’s artwork depicts a sunset at Camp Marie in Leonardtown, Maryland, home of the local MDA summer camp.  Mathew’s hobbies included music and writing poetry.  He collaborated over the Internet with a writer from Wisconsin and together they published a work of poetry called “The Move.”

Seascape

Russell created his painting at age 15 at an MDA-sponsored children's art workshop.  While a student at Lackland Air Force Base High School, he enjoyed playing junior varsity basketball.

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