Research News: First-Ever Gene Therapy for DMD Is Approved

Supporters like you laid the foundation for today’s breakthroughs

It’s one of the most exciting breakthroughs in the history of treating neuromuscular diseases: an FDA-approved gene therapy that could slow or even halt Duchenne muscular dystrophy (DMD).

And it wouldn’t exist without Muscular Dystrophy Association (MDA) supporters like you!

DMD is the most common form of muscular dystrophy in children, mostly affecting boys beginning between ages 2 and 3. It’s caused by a broken gene that’s supposed to produce dystrophin, a protein that’s like a shock absorber for muscles. Without it, muscles are fragile and break down.

Advances in care and treatment, including many from MDA-funded researchers, have radically improved the lives of people with DMD. Still, to really attack the root causes of DMD, we needed the kind of gene therapy that has already reshaped the treatment of diseases like spinal muscular atrophy (SMA).

Gene therapy uses modified viruses to carry working copies of the broken gene into the body, where the working genes can produce proteins that are missing. By replacing damaged genetic information, gene therapy can significantly reduce the effects of some neuromuscular diseases.

DMD challenged researchers because the dystrophin gene is too big for the virus used most commonly in gene therapy to carry. But several research teams have developed a “micro” version of the gene, which has led to the FDA’s historic approval of ELEVIDYS!

Today, for the first time in history and the first time in medicine, we have therapies that can stop some neuromuscular diseases and slow many others. And you help make it possible.

MDA supporters funded the research that launched the entire field of genetic medicine with the discovery of the dystrophin gene’s role in DMD. As you continue your partnership with MDA, you help researchers push forward every single day toward even more breakthroughs. Thank you!

New ALS Treatment Is the First of Its Kind

Because of you, new treatments are coming faster than ever

In April, the FDA approved the fourth drug to treat amyotrophic lateral sclerosis (ALS) since 1995 and the second in less than a year — an incredible mark of progress. And the new drug, Qalsody, has one key difference: It’s the first to target a genetic cause of ALS.

You may know ALS is Lou Gehrig’s disease. It destroys the motor neurons the brain needs to move muscles. Unable to move, the muscles deteriorate. Researchers know that about 10% of people develop ALS because of mutated genes, including one variety called SOD1. This mutation causes toxic protein to build up in the muscles.

Qalsody showed real benefit in reducing the toxic protein and other markers of this variety of ALS. Researchers hope this means that Qalsody can slow down the loss of muscle function and strength. Despite many advances, most people with ALS live only three to five years after diagnosis, so every treatment that can slow its progress is a win.

As the first treatment for a genetic cause of ALS, it also opens the door for new research into other varieties of ALS. And excitingly, Qalsody is now being studied in people who have the SOD1 mutation but haven’t developed symptoms yet, to see if it can prevent ALS altogether.

MDA — thanks to supporters like you — was among the first to invest in the initial studies that proved this approach had promise. “It’s gratifying to see a strategy that we and other organizations funded substantially in earlier stages maturing into a real solution for people living with this genetic form of ALS. It represents the fulfillment of the promise of effective genetic medicines for ALS patients and their families,” says Sharon Hesterlee, Ph.D., Chief Research Officer, MDA.

Visit mda.org/als to learn more about ALS and today’s research efforts!

Advocacy Update

Tell Congress to make air travel more accessible

“I had not flown in 15 years, but flew to Washington DC for the MDA Hill Day to help advocate for more accessible air travel. Feeling deprioritized and dehumanized the entire flight there and back, I came away even more disillusioned about air travel for wheelchair users than I already was.” —Mindy Henderson, MDA Editor-In-Chief, Quest Media

Congress must reauthorize the Federal Aviation Act (FAA) by September 30, 2023, to avoid big interruptions in air travel. This “must pass” bill is our opportunity to enact vital measures that will improve air travel for people living with disabilities.

This year, we’re hoping Congress will pass the following reforms during FAA Reauthorization:

1. 1. Making airplanes more accessible
2. 2. Ensure the safety of air travelers with disabilities
3. 3. Minimizing damage to wheelchairs and other mobility devices
4. 4. Holding airlines accountable for broken, damaged, or lost wheelchairs and violations of passenger rights
5. 5. Giving the disability community a seat at the table

   Your legislator needs to hear from you! Urge Congress to finally improve air travel for people living with disabilities!

   Visit mda.org/AirTravel and use the simple tools to send your message today.

Mark Your Calendar

MDA events are a fun way to raise funds and awareness to empower families living with neuromuscular diseases. We truly appreciate the event participants, dedicated volunteers, and generous donors who make each event a success.

Here are some upcoming events open to the public. For a complete list, more information, and to register, visit mda.org/events.

The MDA Art Collection Is Online!

All across our country, talented artists who are living with muscular dystrophy, ALS, and related neuromuscular diseases are creating unique and beautiful artwork. We are privileged that so many share their artwork with MDA.

You can enjoy the full collection online at mda.org/art.

These more than 400 original works by artists from all 50 states, the District of Columbia, and Puerto Rico prove that physical disability is no barrier to creativity. Artists from ages 2 to 84 used a wide variety of media from traditional paints to digital designs, to collages with corn, to paint applied with wheelchair wheels and human feet.

The subject matter ranges from self-portraits to landscapes and from still lifes to outer space fantasies. Crucially, the art also showcases each artist’s pride in their experiences, including those as people with disabilities. That makes the collection a powerful voice to communicate the culture of disability.

Visit the collection today to be delighted, amazed, and inspired!

August Is National “Make a Will” Month: Are You Ready?

Next month, August, is National “Make a Will” Month. It’s a great annual reminder to update your will if needed and create a will if you don’t have one already. A will is an important estate-planning document that can help you transfer your assets with ease, but despite this, nearly two-thirds of American adults don’t take the time to create one.

Here are a few more benefits of having a will:

• It allows you to distribute your property and protect your loved ones after you pass away.
• It can provide peace of mind for you and your family.
• You can plan for those in your care (e.g., naming a legal guardian for your children or pets).
• It may prevent family conflict.
• It can eliminate confusion over assets.
• It can ensure that your assets go to the people you want to have them.
• It can help you build a lasting legacy.
• You can use your will to benefit MDA (see mdalegacy.org).

Whether you have $10 worth of assets or $10 million, creating a will is a good idea to give your family more guidance after you pass. Consider creating or updating your will this August for National “Make a Will” Month!