Thursday night, June 2, 2022, individuals from the Muscular Dystrophy Association (MDA), along with local volunteers, MDA families and MDA partners descended on Hotel ZaZa in Memorial City, Houston, Texas for what was truly a magical night. The evening consisted of a never-ending photo-op station, lots of conversation and reconnecting with friends, food AND the . . .
Researchers at the University of California, Davis Neuromuscular Research Lab are seeking individuals with or without neuromuscular disease (ages 2 years or older) to participate in a research study to test the feasibility of using a wearable gait sensor to collect data as part of a new outcome measure for use in future clinical trials. This . . .
Drug companies are developing more therapies for rare disease patients — and doing it faster — than ever before. But when it comes to enrolling minorities in clinical trials, the US track record is lagging behind. That subject was the focus of a March 15 session during the 2022 MDA Clinical & Scientific Conference in . . .
On April 5, an international research team, headed by Michela Guglieri at Newcastle University and Robert Griggs at the University of Rochester Medical Center, announced positive results at the American Academy of Neurology (AAN meeting) in Seattle, WA from the phase 3 FOR DMD study of corticosteroid dosing regimens for treatment of people with Duchenne . . .
When Yuliya Matyushenko was 12 years old, the Paris-based rare disease organization Eurordis featured her on a poster promoting Rare Disease Day 2015. In late February, the young woman with spinal muscular atrophy (SMA) was hiding in a bare concrete basement in Kharkiv as Ukraine’s second-largest city, located only 35 kilometers from the Russian border, . . .
Today was the second full day of sessions of 2022 MDA’s Clinical & Scientific Conference. The three major themes of today’s sessions were (1) therapeutic strategies to treat NMDs, (2) clinical trial design and implementation, and (3) optimizing patient care. Highlights Mitochondrial interventions: small molecule and gene therapy Carlos Moraes, PhD, from the University of . . .
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 . . .
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 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 . . .