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.
MD and Your Heart Monitor: 6 Things I Wish I'd Known
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 ...
Sarepta to buy Arrowhead’s muscular dystrophy treatment programs
Dec 3, 2024 · ARO-DUX4 is being tested for facioscapulohumeral muscular dystrophy (FSHD) and ARO-DM1 for myotonic dystrophy type 1 (DM1). Both were designed using Arrowhead’s targeted RNAi molecule (TRiM) platform. “We look forward to embarking on this partnership with Arrowhead, having been impressed with their scientific capabilities in developing a potentially best-in-class approach to siRNA,” Doug ...
PPMD 2024: Gene therapy trials recruiting patients around world
Jul 5, 2024 · Sarepta’s Elevidys is 1st FDA-approved DMD gene therapy. A few weeks ago, Sarepta Therapeutics’ Elevidys (delandistrogene moxeparvovec-rokl) became the first gene therapy to win full approval from the U.S. Food and Drug Administration (FDA) for DMD patients 4 and older who are ambulatory (able to walk). The FDA also okayed Elevidys for nonambulatory patients in the same age range, though ...
SOLVE FSHD offering $10M toward research on FSHD treatments
Dec 10, 2024 · Finding a treatment for FSHD. Like other forms of muscular dystrophy, FSHD is marked by muscle weakness that worsens over time. FSHD usually most severely affects the muscles around the face, shoulder blades, and upper arms, but it also can affect other muscles, and 1 in 5 people with the disease will be unable to walk by the time they are age 50.
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.
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
Capricor now seeking FDA approval of DMD therapy deramiocel
Oct 10, 2024 · After three years of treatment in the extension study, data showed sustained benefits in heart and arm function compared with the decline seen among untreated patients.. Capricor now is sponsoring the Phase 3 HOPE-3 trial (NCT05126758), which is recruiting a similar patient population to that of HOPE-2 at sites in the U.S. The trial is primarily assessing the effects of deramiocel on arm and ...
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.
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.