Stay informed. See MDA updates on COVID-19

Latest Editions

Recent Quest Articles

Outside the Lab

Michael Lo Sapio, father of Mikey, 7, and Reid, 5, proudly admits that he’s pushy. Both his boys live with Duchenne muscular dystrophy (DMD), which causes progressive muscle degeneration and weakness beginning in early childhood. People with DMD typically live to their late teens or early 20s.These facts are grim, but being pushy means not accepting the statistics without a fight. For Michael — and many people living with neuromuscular diseases — one way to push back on the numbers is by participating in research.

Read More

What’s the Toy Story?

Barbies were a favorite toy of young Jessica Hetzel, despite the fact that the dolls looked nothing like her. It took until February 2019, when Jessica was 17, for Mattel to release a Barbie in a wheelchair. “I’m not playing with Barbies these days,” says Hetzel, who has spinal muscular atrophy (SMA). “But I was excited to see they finally did it.”

Read More

More Than a Game

A typical weekend day for me starts off with my partner or caregiver feeding me breakfast (Cheerios with milk on the side) and ends with me falling asleep to an episode of “Cutthroat Kitchen.” In between these moments, I game with friends from all over the country. Gaming has been part of my life since the 1980s. I grew up playing on all the major gaming systems and had a constant slew of competitors in my brother and neighborhood friends. But as congenital muscular dystrophy (CMD) affected my body over the years, and as game controllers became more complex, I started having trouble keeping up with my peers.

Read More

Wheels Like Mine

I am a mom. I have spinal muscular atrophy (SMA). My son has SMA, too. Because SMA is a genetic disorder, these statements may not seem surprising. It wouldn’t be all that strange for a person with SMA to pass on their condition to their child. But that is not our story. Our journey to family is more convoluted and, dare I say, even more beautiful than most would guess. Ours is a story of love and adoption and beauty where others see tragedy.

Read More

The Ups and Downs of Accessible Air Travel

Cory Lee, who lives with spinal muscular atrophy (SMA), has explored 32 countries in the past five years. While traversing the globe in his 300-pound power wheelchair, Lee writes about accessible travel on his blog curbfreewithcorylee.com, which has garnered thousands of followers. The No. 1 topic people ask him about is air travel.

Read More

Targeting Genes

Until the 1980s, little was known about the cause of any neuromuscular disease. In 1986, MDA-supported researchers identified a single gene on the X chromosome that leads to Duchenne muscular dystrophy (DMD) when it is mutated (flawed). In 1987, the protein associated with that gene was identified and named dystrophin. Lack of the dystrophin protein in muscle cells causes them to be fragile and easily damaged.

Read More

Guiding Treatment for Myotonic Dystrophy

Myotonic dystrophy (DM) is the most common form of muscular dystrophy in adults. This is a complex disease, affecting not just the muscles, but nearly every other organ system in the body. The signature manifestation is myotonia, an inability to consciously relax the muscles, coupled with progressive muscle weakness.

Read More

Pushing Limits

After six long days, I’m tired. It’s the middle of the night, I’m wearing five layers of clothing and I know I have several more hours to go in the frigid air before I reach the 19,341-foot summit. “Mom believes in you. Dad believes in you. All of your friends and family believe in you. You are strong,” are the words that echo through my mind with every step.

Read More

The State of Gene-Targeted Therapies

The first full day of MDA’s Clinical & Scientific Conference included a highly anticipated session that took a deep dive into gene-targeted therapies. Experts discussed how gene-replacement, gene-silencing and gene-editing therapies are being studied and tested for treating patients with a variety of neuromuscular diseases (NMDs).

Read More

Ongoing Clinical Trials

During the second day of the 2019 MDA Clinical & Scientific Conference, 14 scientists and clinicians shared exciting updates from the pipeline of clinical trials. Below are highlights from the session.At the time of the conference, Zolgensma (onasemnogene abeparvovec-xioi, or AVXS-101) was under review by the U.S. Food and Drug Administration (FDA). In May, the FDA approved the drug as a therapy for the treatment of children younger than age 2 with spinal muscular atrophy (SMA).

Read More

Manage your Quest subscription

 

 

MDA Resource Center: We’re Here For You

Our trained specialists are here to provide one-on-one support for every part of your journey. Send a message below or call us at 1-833-ASK-MDA1 (1-833-275-6321). If you live outside the U.S., we may be able to connect you to muscular dystrophy groups in your area, but MDA services are only available in the U.S.

Request Services