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.

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.

6 days ago · Corticosteroid at low dose may help with muscle damage, inflammation. Once a week treatment with a corticosteroid was seen to lower some biomarkers of muscle damage and inflammation, helping to improve muscle function in people with some forms of muscular dystrophy, according to a pilot study in the U.S. Prednisone was …

Dec 17, 2024 · “This landmark study presents compelling biomarker data and promising signals that suggest the potential for functional stabilization with administration of sevasemten,” Craig McDonald, MD, a principal trial investigator and professor at the University of California Davis, said in a company press release.. Edgewise scientists will join Parent Project Muscular Dystrophy to …

Nov 25, 2024 · The power to influence or even direct people’s behavior or the course of events is part of a typical definition of “control.” When living with a chronic illness, as I am with limb-girdle muscular dystrophy, our desire to control the course of events can be a creative balancing act.We hope it’s like riding a wave slowly and smoothly, ending on warm sand.

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

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 ...

Jul 3, 2024 · To get around this, some gene therapies, such as Elevidys (delandistrogene moxeparvovec-rokl), which was recently authorized in the U.S. most DMD patients ages 4 and older, deliver a gene that encodes mini-dystrophin, a shortened, but functional version of the protein.. Kinea Bio is pioneering a strategy that takes advantage of molecules called inteins to deliver a larger version of dystrophin ...

Aug 22, 2024 · Elevidys became the first and only gene therapy available for DMD patients after the U.S. Food and Drug Administration (FDA) conditionally cleared it in June 2023 for boys, ages 4-5, who were still able to walk. Hiram Secrist and other eligible boys were dosed just a few weeks later.. The boy’s mother, Kristen Secrist, said …

Jun 28, 2023 · Approved exon-skipping therapies Exondys 51. Exondys 51 (eteplirsen) was developed by Sarepta Therapeutics as a treatment for DMD patients with mutations amenable to exon 51 skipping. That’s about 13% of all people with DMD. The FDA granted Exondys 51 accelerated approval in 2016. The therapy is administered once weekly via an infusion into the bloodstream that takes 35 minutes to one hour.