Quest Magazine

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

DMD/BMD Research: Ataluren Results Disappointing

The biopharmaceutical firm PTC Therapeutics announced March 3 that ataluren, its experimental drug for certain forms of Duchenne (DMD) and Becker (BMD) muscular dystrophy, although safe and well tolerated, failed to meet its primary end point within the 48-week duration of the phase 2b trial. That end point was an improvement in how far boys with DMD or BMD could walk in six minutes.

Gene Therapy Rescues Mice with SMA

Scientists at four U.S. institutions have successfully used gene therapy to treat very young mice with a disease resembling severe spinal muscular atrophy (SMA).  Study results were published online Feb. 28, 2010, in the journal Nature Biotechnology.

A Quicker Trip from ‘Microscope to Marketplace’?

The National Institutes of Health (NIH) and the U.S. Food and Drug Administration (FDA) have announced plans to establish a Joint NIH-FDA Leadership Council, with the goal of shortening the time it takes to move promising therapies through the regulatory process and into the hands of waiting patients.

SMA Therapeutics: Changing the Code

 A process called trans-splicing has been shown to increase levels of a needed protein in mice with a disease resembling severe human spinal muscular atrophy (SMA), says a research team at the University of Missouri-Columbia, whose findings were published Jan. 6, 2010, in the Journal of Neuroscience.

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