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 . . .
Researchers at Newcastle University are seeking adults living with general pain or with myotonic dystrophy (DM) to participate in a survey to learn about the ability of individuals affected by these conditions to self-manage pain and to determine how this affects overall well-being. This study involves a 15-20 minute online survey. To be eligible, individuals must . . .
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 . . .
Becker muscular dystrophy (BMD) is a rare genetic disorder involving mutations of the dystrophin gene. BMD exhibits similar signs and symptoms to another condition, Duchenne muscular dystrophy (DMD), which is caused by different mutations in the same gene. Although both conditions affect skeletal muscles used for movement, as well as heart muscles, they differ in . . .
On Dec. 17, the US Food and Drug Administration (FDA) granted approval to efgartigimod (Vyvgart) for the treatment of generalized myasthenia gravis (gMG) in adults who test positive for the anti-acetylcholine receptor (AChR) antibody. To date, Vyvgart is the second disease modifying drug approved to treat gMG. Vyvgart will be made available in the United States . . .
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. . . .
Researchers at Worrell are seeking adolescents (ages 12 – 17) with the following conditions: neuromyelitis optica spectrum disorder (NMOSD), myasthenia gravis (gMG), myelin oligodendrocyte glycoprotein antibody disease (MOG-AD), or autoimmune encephalitis (AE), to participate in a research study to test the safety and efficacy of a new syringe device being developed to administer a drug to . . .
Researchers at the Hospital for Sick Children in Toronto, Canada are seeking young adults living with muscular dystrophy to participate in a survey about their interest in and experiences receiving genetic information. Participants will be required to complete a 10-15 minute online survey that collects largely quantitative data, as well as some open-ended responses. After completing . . .