Quest Magazine

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.

The Play’s the Thing

Surrounded by loaves of bread, 12-year-old Andrew Longwell looks like a real baker. The chef’s hat helps, too.

The sixth-grader from Rochester, N.Y., completes the picture with his voice. He sings a charmingly youthful rendition of “Who Will Buy?” one of the songs in the musical “Oliver!” He and other students are performing the show at the Greece Athena Middle School Theatre in Rochester for two weeks starting Feb. 26.

Wasn’t gonna do it

Building New Muscle Requires Controlled Damage

Remodeling a building usually requires some degree of dismantling before new construction can begin. That principle, it now seems, also may apply to the remodeling of the body's cells. 

Scientists at Ottawa Hospital Research Institute and the University of Ottawa have found recently that it applies to muscle cells, which can only develop from a stemlike state into mature muscle fibers after a certain amount of their DNA has been disassembled and then rebuilt. 

About the new findings 

SMA Research: Strengthening the Junctions

MDA-supported researchers at the University of Ottawa and Ottawa Hospital Research Institute (OHRI) have identified a biological pathway that may prove useful in developing treatments for spinal muscular atrophy (SMA)

Causative Gene Mutations ID'd for Two Muscle Diseases

An MDA-supported, multinational team of researchers from Canada and Europe has identified specific mutations in the anoctamin 5 (ANO5) gene on chromosome 11 that can cause type 2L limb-girdle muscular dystrophy (LGMD2L) and type 3 Miyoshi myopathy.

Cardiomyopathy in Becker MD

This story was updated Oct. 6, 2010.

In many forms of muscular dystrophy, including Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), weakness and degeneration of the cardiac muscle can be a major part of the disease, and one that frequently shortens life.

CMD, Form of MM Qualify for Speedy Decision on S.S. Benefits

The Social Security Administration (SSA) announced today that 38 more diseases have been added to its Compassionate Allowances list, including four forms of congenital muscular dystrophy (CMD) and Leigh syndrome, a form of mitochondrial myopathy.

New Muscle Stem Cell Found in Mice

MDA-supported scientists in France have identified a previously unknown type of muscle stem cell located in the spaces between muscle fibers in mice. They say the new cells, dubbed "PICs," may play at least as important a role in muscle regeneration and repair as satellite cells, which have been recognized as stemlike cells in muscle since the 1960s. As such, they could have implications for treatment of muscular dystrophies.

DMD, BMD: Tests Start of Experimental Utrophin Drug

 A small, orally administered molecule designed to increase production of the muscle protein utrophin, is being tested in healthy volunteers, according to its developer, BioMarin Pharmaceutical of Novato, Calif.

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