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4 days ago · Stuck in limbo with both accessible vans out of commission. I live in rural Nebraska with my husband and our seven children. Three of them — Max, 19; Rowen, 15; and Charlie, 14 — live with Duchenne muscular dystrophy (DMD).
The link between DMD and gastrointestinal issues – Muscular...
Jul 15, 2024 · Prevalence. Many people living with DMD will have a GI issue at some point. It becomes more common as the disease progresses. Williams says he sees an average of six children and teens a month in the pediatric clinic at National Children’s Hospital who have been screened for help with a GI problem.
Gene therapy BB-301 at low dose helping OPMD patients swallow
Oct 15, 2024 · The trial, cleared by U.S. regulators last year, is evaluating BB-301’s safety and its effects on swallowing in OPMD patients.In its Phase 1b part, patients are being treated sequentially at three different dose levels in order to select an optimal gene therapy dose to be tested during the trial’s Phase 2a part.
Sevasemten lowers markers of muscle damage in BMD: Trial
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 …
Eye Problems | Muscular Dystrophy News
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Taking control when living with limb-girdle muscular dystrophy
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.
FDA clears Phase 2 study of exon skipping therapy for DMD
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.
Duchenne muscular dystrophy: Exercise and physical therapy
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
NS Pharma DMD treatment gets FDA rare pediatric disease status
Sep 12, 2024 · NS-050/NCNP-03 is an antisense oligonucleotide, a short strand of genetic material. The therapy skips over exon 50, an approach amenable for about 4% of the DMD population.According to NS Pharma, NS-050/NCNP-03 aims to enable production of a functional yet slightly shorter dystrophin protein, which is expected …
Adaptive aids for Duchenne muscular dystrophy – Muscular …
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.