2022 Clinical & Scientific Conference March 14 Highlights

Today was the first full day of sessions of MDA’s 2022 Clinical & Scientific Conference. For the first time since the start of the COVID-19 pandemic, clinicians, scientists, policymakers, nonprofit, and industry leaders are convening, primarily in-person, for a dynamic and informative five days in Nashville, TN. With more than 975 in-person and 490 virtual . . .

Read More

Clinical Trial Alert: Phase 1b/2a Study of WVE-N531 in a Subset of People Living with DMD

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 . . .

Read More

Research Study Alert: Survey of Pain Self-Management and Well-Being in People with General Pain or with Myotonic Dystrophy

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 . . .

Read More

Simply Stated: What is Duchenne Muscular Dystrophy?

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 . . .

Read More

Simply Stated: What is Becker Muscular Dystrophy?

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 . . .

Read More

FDA Approves argenx’s Vyvgart for Treatment of gMG

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 . . .

Read More

Research Study Alert: Survey for Siblings of Individuals with DMD about Disclosure and Communication within the Family

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. . . .

Read More

Research Study Alert: Study of New Device for use in Adolescents Living with Autoimmune Neuromuscular Conditions

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 . . .

Read More

Research Study Alert: Survey for Young Adults with Muscular Dystrophy

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 . . .

Read More

Research Study Alert: Natural History Study in Males with BMD

Researchers at the University of Florida are seeking adult males living with Becker muscular dystrophy (BMD) to participate in a three-year natural history study. The objective of this observational study is to gain a better understanding of the course of BMD and to identify biomarkers for BMD. This study does not involve an intervention. Enrolled participants will . . .

Read More