Laura Hagerty, Ph.D. As we settle into 2016 and face a fresh new year full of hope and optimism, much of the focus of our DMD community remains on the exon skipping therapy at the regulatory review end of the development pipeline: Sarepta Therapeutics’ eteplirsen. In November we saw our first advisory committee review for . . .
Kasey Bennett loves football. In fact, it was during one of his games that his parents noticed there was something different about their young quarterback. “He couldn’t keep up with the other kids,” says his father. Soon after, Kasey was diagnosed with DMD. The disease has taken him out of the game but it hasn’t . . .
Zane Jacobs is a “wild and crazy” 7-year-old who “loves to get dirty and cause absolute chaos.” Though he is affected by DMD, he and his family are determined to make the most of their time together, whether that’s playing football in the front yard or swimming at the waterpark. When not horsing around with . . .
At MDA, we are beginning 2016 with families at the heart of our mission – literally. The heart in the middle of our refreshed logo symbolizes the kids and adults living with muscular dystrophy, ALS and related life-threatening diseases we fight for every day. We also begin the year with new offerings and enhancements to . . .
Investigators are seeking participants for a new phase 2 clinical trial, sponsored by Cytokinetics, to test the investigational drug CK-2127107 in people with types 2, 3 or 4 spinal muscular atrophy (SMA). CK-2127107 is a fast skeletal muscle troponin activator, designed to increase the ability of muscle to contract by sensitizing it to calcium. In . . .
It’s Christmas baking day today. Actually, the preparations started a few days ago, with a shopping trip, of course. Then, last night, Katherine helped me make a batch of Shortbread Cookies using my grandmother’s recipe. We also made a batch of pastry dough for the mince tarts and butter tarts planned for today. So now . . .
People may know TNT’s Ernie Johnson as the NBA host who shares the stage with pros like Charles Barkley and Shaq. But it’s what goes on behind the scenes that makes him a star in our eyes. This touching story gives us a glimpse into Johnson’s life off-screen as a devoted father of six who . . .
For 42-year-old Warner Smith, treatments and cures for ALS can’t come fast enough. Like any doting father, Warner wants to be around for all his young daughter Carlee’s milestone moments. He wants to teach her to drive, see her go to prom and walk her down the aisle at her wedding. Every moment is precious . . .
The Muscular Dystrophy Association (MDA) today announced that Jiffy Lube has raised more than $1 million during the fourth annual MUSCLE UP!SM campaign to help kids and adults with muscular dystrophy, ALS and related muscle-debilitating diseases live longer and grow stronger. “Each day, kids and adults with neuromuscular diseases are losing their ability to do . . .
Diane Merry, associate professor at Thomas Jefferson University in Philadelphia, is working to identify therapeutic opportunities to promote normal androgen receptor function while preventing the toxic effects of polyglutamine expansion in spinal-bulbar muscular atrophy (SBMA). With GlaxoSmithKline, Merry will test small molecule compounds that activate an enzyme called SIRT1 in cell and mouse models of . . .