Quest Magazine

$ for College

Ten $1,000 scholarships for high school seniors and college students whose parents have disabilities are being offered nationwide by the National Center for Parents with Disabilities and their Families (NCPDF).

The scholarships will be awarded in fall of 2009. Applications must be completed and postmarked by March 16.

NCPDF is the national agency of Through the Looking Glass, a San Francisco-area philanthropic organization.

DMD/BMD Reseach: Minidystrophin Gene

A promising “minidystrophin gene” that restores normal muscle force to skeletal and diaphragm muscles in mice with a disease resembling Duchenne muscular dystrophy (DMD) seems to be only partially effective at restoring strength and function to heart muscles.

DMD Research: Another Dystrophin Stand-In?

Delivering the gene for the missing dystrophin protein to the muscles of children and young men with Duchenne muscular dystrophy (DMD) is one of several therapeutic avenues under intense investigation in this disease. However, the strategy has technical drawbacks, and some experts worry that introduction of a previously absent protein could provoke a dangerous immune response.

CMD Research: Promising Leads

Recently published findings from two independent groups have suggested possible treatment pathways for the merosin-deficient and integrin-deficient forms of congenital muscular dystrophy (CMD).

Doxycycline fights cell death and lessens disease severity in merosin-deficient mice

Flying High ... From a Wheelchair

Flying automobiles … flying lawnmowers … flying doghouses … where’s this madness going to end?

If Chris “Lucky” Carnes has his druthers, the sky’s the limit.

For the past four years, the 33-year-old from Chase City, Va., has been busy practically every weekend with the rapidly growing hobby/sport of model aviation.

MMD Research: Drug Development?

The identification of small molecules that can block the genetic defect that causes type 1 myotonic dystrophy (MMD1, or DM1) may be the first step toward developing a new drug treatment for the disease, say researchers at the University of Rochester (N.Y.) Medical Center (URMC).

The abnormality that underlies MMD1 is a stretch of genetic material derived from DNA on chromosome 19 that contains more than the usual number of a repeating chemical sequence known as a CUG (cytosine, uracil, guanine) triplet repeat.

Recreational Opportunities for Wheelchair Users

Like many people who served in the U.S. Marine Corps, Ray Brown, 59, sees no difference between then and now. “I’m still a Marine,” he attests firmly.

That dogged determination no doubt has been largely instrumental in the success of Wheelin’ Team 457. It’s an organization Brown formed in 2003 to provide recreational opportunities for people with disabilities who use wheelchairs.

SMBA Research: Can This Cell Be Saved?

A defense mechanism called “autophagy” that neurons (nerve cells) use to protect themselves from dangerous misfolded proteins may hold the key to developing treatments for spinal-bulbar muscular atrophy (SBMA, or Kennedy disease) and perhaps similar neurodegenerative diseases, new research shows.

Speeding the Course of Clinical Trials

Trial Results in DMD Heralded as 'Proof of Concept'

The Dec. 27 issue of the prestigious New England Journal of Medicine featured encouraging results of a phase 1 clinical trial of an exon skipping compound in four boys with Duchenne muscular dystrophy (DMD). Preliminary findings from this trial, conducted in the Netherlands, were announced in May (see Progress Reports).

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