MDA 2012 Conference Report: Targeted Therapies

MDA 2012 Conference Report: Targeted Therapies

In this second report on MDA's Clinical Conference, updates on experimental therapies in development for six neuromuscular diseases are described

MDA Funds Testing of Anti-Inflammatory Compounds for DMD

MDA Funds Testing of Anti-Inflammatory Compounds for DMD

MDA awarded $120,000 to Catabasis Pharmaceuticals to test two anti-inflammatory compounds, CAT-1004 and CAT-1040, in the mdx mouse model of Duchenne MD

MMD1: 'Invasive' Approach to Cardiac Management Improved Survival

MMD1: 'Invasive' Approach to Cardiac Management Improved Survival

Adults with MMD1 and minor cardiac conduction defects benefit from invasive testing and possible pacemaker insertion

Eteplirsen Led to Dystrophin Production in U.S. DMD Trial

Eteplirsen Led to Dystrophin Production in U.S. DMD Trial

Twenty-four weeks of treatment with the exon-skipping drug eteplirsen resulted in significant increases in dystrophin production

AAN Releases Guidelines for IVIG in Some Neuromuscular Diseases

AAN Releases Guidelines for IVIG in Some Neuromuscular Diseases

The American Academy of Neurology has released guidelines for the use of intravenous immunoglobulin, which modulates the immune system in MG, LEMS and myositis

SMA Disease Course May Be Affected by ZPR1

SMA Disease Course May Be Affected by ZPR1

Zinc finger protein 1 (ZPR1) appears to be a 'protective modifier' of spinal muscular atrophy, making it a potential target in the development of therapies

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A challenging and multifaceted disease

posted on April 9, 2012 - 12:05pm
As far back as Carla Licon can remember, her mother had difficulty opening jars and walking long distances. Licon’s mother wore ankle braces, and she also had an unusual symptom known as "myotonia," the inability to relax muscles, such as a clenched hand, at will. Licon, who is 31 and lives in Victoria, Texas, thinks these symptoms started when her mother was in her 20s. Later, her mother’s...

Getting on the level

posted on April 9, 2012 - 12:04pm
Whether facing a 2-inch-high curb or a 3-foot-tall porch, the results for a person in a wheelchair can be exactly the same: no passage. The way around both these barriers is, of course, a wheelchair ramp, though they will be decidedly different. The first might be a portable, foldable aluminum device, while the second might be a large, permanent structure made of concrete or galvanized steel....

Iconoclast and author Mike Murphy wants a revolution in the way characters with disabilities are portrayed

posted on April 9, 2012 - 12:03pm
I love movies. Always have, always will. I pumped my fist in the air (metaphorically) when Captain America beat the snot out of Red Skull, held my breath (also, metaphorically) while Frodo and Sam scaled Mount Doom, and teared up when the starship Enterprise first appeared in the latest Star Trek movie.

Students hop to help fight muscle disease, while learning core values of awareness, acceptance and assistance

posted on April 9, 2012 - 12:02pm
Hopping isn’t just for bunnies. Each year thousands of preschools, day care centers and elementary schools across the country are taken over by swarms of hopping students, all doing their part to help children with neuromuscular disease.
posted on April 9, 2012 - 12:01pm
Carrot 3 car seat Convaid offers a restraint system made specifically for children with special needs. The Carrot 3, a top-selling car seat from Europe, is designed to promote good positioning and safety. It has built-in growth functions (plus modular extensions and accessories) that accommodate children from ages 3 to 15, between 30 and 108 pounds. Conforming to the natural contours of the...

A big sports fan with a bigger heart, this 11-year-old is ready to speak out for MDA

posted on April 9, 2012 - 12:00pm
Bryson Foster dreams big. No, make that B-I-G. But those with big hearts usually do. "Even if you dream small, those small things can turn into big things in the end," says Bryson, with wisdom beyond his 11 years. In true sports vernacular, he adds: "Go big or go home." So, Bryson continues to dream about one day being the starting quarterback for his beloved Cincinnati Bengals, or a professional...
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