Quest Magazine

DMD Research: Potential Heart Saver

A synthetic compound that seals cellular membranes has been found to stop the progression of heart-muscle destruction in dogs with a disease closely resembling human Duchenne muscular dystrophy (DMD), MDA-supported researchers have found.

Lumizyme on the Horizon

Biotechnology company Genzyme, of Cambridge, Mass., expects the U.S. Food and Drug Administration (FDA) to rule by June 17, 2010, on Genzyme's application to market its laboratory-developed enzyme Lumizyme (alglucosidase alfa) in the United States.

Lumizyme and its near-twin, Myozyme, replace the acid maltase enzyme deficient in people with Pompe disease (acid alpha-glucosidase deficiency or acid maltase deficiency)

SMA Research: Saving Shortened SMN Protein

A research team at the University of Pennsylvania in Philadelphia has characterized the mechanism responsible for rapid decay of the survival of motor neurons (SMN) protein that is encoded by the human SMN2 gene and which plays a key role in a variety of therapeutic strategies under development for spinal muscular atrophy (SMA)

Rising to the Challenge

In the documentary “Waiting to Rise,” Rob Roozeboom’s small son asks him, “Dad, why do you walk like that?”

“Because I have muscular dystrophy, son,” he answers.

The boy is unconvinced. “Penguins walk like that,” he observes, “but they don’t have muscular dystrophy.”

At this stage of his life, Roozeboom, 33, can laugh uproariously at that assessment. But as a boy himself, and later as a teen, his disability was no laughing matter.

Cash and Counseling Takes Hold

Cash and Counseling, a government program that gives Medicaid recipients in 15 states direct control over their personal-care spending, has been so successful that a majority of states are now adopting it or similar programs. A new report tells why and how.

Get to Work!

The Social Security Administration (SSA) and other service providers have launched several new Web sites for people with disabilities who want to work.

The Choose Work site contains first-person accounts by people who have used the SSA's work incentives; videos, including a seminar on work incentives; help finding local resources; and more. The site can be found at http://www.choosework.net/.

Gene Therapy Book by MDA Grantee

MDA grantee Dongsheng Duan has published a book on the latest advances in gene therapy for muscle disease, particularly muscular dystrophies.

Duan, a professor of microbiology and immunology at the University of Missouri, served as editor of the book Muscle Gene Therapyand co-authored two of its 15 chapters. Duanis currently researching gene transfer therapy in dogs.

About the new book

ALS SOD1 Trial: A ‘Watershed Moment’

Isis Pharmaceuticals of Carlsbad, Calif., has begun a phase 1 clinical trial of its experimental compound ISIS-SOD1-Rx in people with familial (inherited) ALS caused by toxic SOD1 protein molecules.

ALS SOD1 Trial: A ‘Watershed Moment’

Isis Pharmaceuticals of Carlsbad, Calif., has begun a phase 1 clinical trial of its experimental compound ISIS-SOD1-Rx in people with familial (inherited) ALS caused by toxic SOD1 protein molecules.

Thinking of College? Think Scholarships

Application deadlines are fast approaching for two scholarships for students with disabilities, offered by two organizations run by people with forms of muscular dystrophy.

Both organizations – Incight and Deshae Lott Ministries -- require applicants to submit documentation of disability, confirmation of acceptance at a qualifying educational institution, and letters of recommendation. More details are available on their Web sites.

Incight
(971) 244-0305
www.incight.org

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