Jan 2, 2025 · Read the story here. No. 8 – PPMD conference highlighted DMD gene therapy and treatments. As part of our coverage of the 2024 Parent Project Muscular Dystrophy (PPMD) Annual Conference, we reported on a session discussing up-and-coming treatments, including gene therapies and approaches to regenerate muscle cells. As the DMD gene that is mutated in people with Duchenne is too big to fit ...

Jun 21, 2024 · Assistive technologies. Advancements in technology have been a great help to people living with DMD, from enhancing accessibility with eye-tracking devices, smart eyeglasses, and sip-and-puff devices, to aiding communication and fostering social inclusion through voice recognition software.

Jan 31, 2025 · MDA honors researcher, patient advocate with 2025 Legacy Awards. The Muscular Dystrophy Association (MDA) has announced its 2025 Legacy Awards, and will honor two individuals who have made pioneering strides toward improving life for people affected by muscular dystrophy.

Apr 18, 2023 · The results showed that dystrophin protein levels increased to a mean of 10.27% of normal in patients treated with the low dose, and to 15.79% of normal in those treated with the high dose.. Motor function was either maintained or showed a trend for improvements, as assessed by the North Star Ambulatory Assessment score.

Jan 23, 2018 · If you’re like me and have been closely monitored by a cardiologist for one reason or another (mine is tachycardia and cardiomyopathy), then you probably have or will have a heart monitor.A Holter heart monitor is a device that is used to record your heart’s activity. You may have to wear it for any amount of time spanning from 24 hours to 30 days, depending on your condition and what your ...

Jun 17, 2024 · A physical therapist knowledgeable about DMD and progressive muscle weakness and other DMD symptoms can create a therapeutic exercise program that safely stretches and strengthens the body. They also can show you how to use assistive devices such as mobility aids and orthotic devices.. Benefits of physical therapy for people with DMD

Dec 10, 2021 · I often see posts in Facebook groups and other places asking about what holiday gift ideas people might have for sons with Duchenne muscular dystrophy (DMD). Duchenne can affect different muscle groups, and patients can experience both muscle weakness and problems with muscle control in performing tasks and activities, so I have to find ways to match their interests with their abilities.

Jul 2, 2024 · PTC Therapeutics plans to soon ask the U.S. Food and Drug Administration (FDA) to approve ataluren as a treatment for Duchenne muscular dystrophy (DMD) caused by nonsense mutations. “We are thrilled to announce that we are moving forward with the submission of a new drug application for ataluren here in the U.S.,” Jonathan Blaize, PhD, medical science liaison at PTC, said at the Parent ...

Jul 4, 2024 · In Duchenne MD, mutations lead to a lack of functional dystrophin, a protein that normally helps protect muscles from damage.That lack of dystrophin causes muscles to accumulate more wear and tear over time, ultimately resulting in DMD symptoms.. Some DMD treatment strategies, such as exon-skipping and gene therapy, aim to target the disease’s root cause by boosting levels of functional ...

Duvyzat (givinostat) is an oral therapy approved in the U.S. for adults and children, ages 6 and older, with DMD. It aims to reduce inflammation and muscle loss in people with DMD, ultimately working to slow disease progression.