Myotonic dystrophy (DM) is a type of muscular dystrophy that affects about 1 in 8,000 people worldwide. The disease is known by several names, including Steinert disease, after the doctor that first described it, and “DM” in reference to its Greek name, dystrophia myotonica. Similar to other forms of muscular dystrophy, DM is characterized by . . .
Researchers at Wave Life Sciences are seeking boys living with DMD caused by gene mutations amenable to exon 53 skipping interventions to participate in a phase 1b/2a clinical trial. This trial will evaluate the safety, effectiveness, and correct dosage of the investigational drug WVE-N531. WVE-N531 is an exon-skipping drug designed to promote skipping over a section . . .
At a time when so much is changing, one thing that remains constant is the Muscular Dystrophy Association’s dedication to providing programs and support to help children and adults with neuromuscular disease live longer and grow stronger. A diagnosis of muscular dystrophy, ALS, or other related neuromuscular disease can bring many challenges to individuals and . . .
As the Muscular Dystrophy Association (MDA) continues our commitment to empowering people living with neuromuscular disease, we are excited to present our 2022 blog series: “Quest for Success”. Success looks different to everyone and this monthly blog will detail the different paths that individuals with neuromuscular disease have taken to reach their potential and the . . .
MDA Summer Camp has been a favorite tradition for our young community for over 65 years. For one week every year, children and teens receive the opportunity to gain critical life-skills, including self-confidence, independence, and self-advocacy, while making lifelong friendships in an inclusive and accessible environment. In 2020, in an effort to keep this popular . . .
At Daytona International Speedway, if you see a silver wheelchair-accessible minivan flash by outside the stadium, it’s shuttling people who need assistance getting around the expansive venue. Philip James “PJ” Nicholoff would be happy knowing that his family donated his beloved van to the speedway, and its back windows display signage honoring him. A big . . .
Duchenne muscular dystrophy (DMD) belongs to a group of rare genetic disorders, known as dystrophinopathies, that are caused by mutations of the dystrophin gene. DMD is characterized by progressive degeneration and weakness of the body’s voluntary muscles, primarily the skeletal muscles that control movement. In later stages, the heart and respiratory muscles may also be . . .
In recognition of International Day of Women and Girls in Science, the Muscular Dystrophy Association (MDA) is honored and excited to highlight the career and accomplishments of Dr. Angela Lek, PhD. International Women and Girls in Science Day, February 11, endeavors to acknowledge and celebrate the invaluable role that women and girls play in accelerating . . .
The individuals and families in our neuromuscular community have always been the heart of MDA’s mission. MDA’s Family Support Team executes a variety of programs that empower individuals and families on their journeys. As another year ends, and the new year is ramping up, the Family Support Team is proud to reflect on services provided . . .
Researchers at Boston Children’s Hospital/Brandeis University are seeking unaffected siblings of boys with Duchenne Muscular Dystrophy (DMD) to participate in a survey about their perspectives on disclosure and communication within the family. The information gained from this study will be used to guide parents on how to best communicate information about DMD to their family members. . . .