Adaptive aids for Duchenne muscular dystrophy – Muscular …
Jun 21, 2024 · Physical therapy or occupational therapy treatments may include sessions with standing frames to maintain muscle, or slings to do transfers from chair to therapy table. The therapist may recommend orthotic devices to correct ankle contractures and scoliosis, among other preventive measures.. Mobility devices. Mobility aids such as wheelchairs, scooters, canes, and walkers enable people with ...
PPMD 2024: Trials due for DMD gene therapies, cell regeneration
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 ...
Capricor to seek deramiocel’s FDA approval for DMD heart disease
Sep 26, 2024 · ‘Novel, first-in-class treatment to patients in need’ “Based on the strength of our cardiac data … we are seeking approval for the cardiomyopathy associated with DMD and will look to expand the label for skeletal muscle myopathy post-approval,” Linda Marbán, PhD, Capricor’s CEO, said in a company press release.. “We believe this is the best path forward to potential approval ...
BBP-418 for limb-girdle muscular dystrophy
Sep 30, 2024 · How does BBP-418 work? LGMD type 2i (LGMD2i), or LGMDR9, is a type of muscular dystrophy caused by mutations in the FKRP gene. FKRP provides instructions for the production of the fukutin-related protein (FRKP), which works to add specialized sugar molecules to alpha-dystroglycan — a process called glycosylation.. Alpha-dystroglycan is part of the dystrophin glycoprotein complex.
Rare pediatric disease designation granted to LAMA2-CMD therapy
Oct 3, 2024 · The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to MDL-101, an epigenetic editing therapy that Modalis Therapeutics is developing for LAMA2-related congenital muscular dystrophy (LAMA2-CMD) — a genetic condition that’s characterized by muscle wasting evident at birth or shortly thereafter.
PPMD 2024: PTC to seek FDA approval of ataluren for DMD
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 ...
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.
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
Regenxbio starts pivotal trial testing DMD gene therapy RGX-202
Nov 21, 2024 · The study’s pivotal part is expected to enroll 30 people with DMD, ages 1 and older, who are able to walk. The only gene therapy approved for DMD so far, Elevidys (delandistrogene moxeparvovec-rokl), is available in the U.S. only for patients ages 4 and older. This clinical trial will be the first DMD gene therapy to include children as young as a year, according to Regenxbio.
Amondys 45 (Casimersen) - Muscular Dystrophy News
Oct 18, 2023 · Amondys 45 is an exon-skipping therapy, which works to skip over certain exons, letting the remaining ones fit together better.This enables a shorter, but still functional dystrophin protein, to be produced. Using Sarepta’s proprietary technology, Amondys 45 directs muscle cells to skip exon 45 in the DMD gene. By allowing some dystrophin to be produced, the treatment is intended to slow ...