Agamree recommended for NHS use in UK, approved in China
Dec 12, 2024 · “The approval of Agamree through the Priority Review Program reflects the government’s high level of attention to the development of rare disease drugs,” said Yan Zhiyu, chairman and CEO of Sperogenix, in a separate press release.. DMD affects more than 70,000 families in China, “and until now, there had been no approved treatment option for …
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.
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.
Sarepta stops development of exon 51-skipping therapy for DMD
Nov 12, 2024 · SRP-5051 was a next-generation successor to Exondys 51 (eteplirsen), an older exon 51-skipping therapy also from Sarepta. Exondys 51 is conditionally approved in the U.S. to treat eligible DMD patients. Both therapies contain a lab-made molecule that masks exon 51 and promotes its removal from messenger RNA, an intermediary …
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 ...
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.
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.
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
PPMD 2024: 4 therapies to slow DMD progression now in trials
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 ...