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 …

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.

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 being part of the historic moment “feels …

Jan 9, 2025 · Gene-editing therapy targets DYSF, whose mutations cause LGMD type 2B/R2. MD refers to a group of disorders characterized by progressively worsening muscle weakness and wasting due to mutations in genes encoding proteins important for muscle health.. Mutations in DYSF, which encodes the production of dysferlin, are the cause of LGMD type 2B/R2.Dysferlin …

Jan 21, 2025 · Prednisone was given once weekly for six months to 19 people with limb-girdle muscular dystrophy (LGMD), a disease type that primarily affects the hips and shoulders, and to one adult with Becker muscular dystrophy (BMD), which most often affects the legs. A study aim was to investigate whether such low-dose treatment provides the benefits of corticosteroids …

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 …

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.

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 to suppress muscle function …

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 …

Jan 16, 2025 · The Phase 1/2 ACHIEVE trial (NCT05481879) will now enroll a new group of participants who will all receive the dose that’s so far shown the most promising clinical benefit. Data from this group — called the registrational expansion cohort — could support a request for accelerated approval of DYNE-101 for DM1 in the U.S. Dyne is also pursuing expedited …