News Archives - Muscular Dystrophy News
FDA OKs trial to test gene therapy candidate in LGMD children. The U.S. Food and Drug Administration (FDA) has given Atamyo Therapeutics the go-ahead to start a Phase 1b clinical trial to test ATA-200 — the company’s gene therapy candidate for limb-girdle muscular dystrophy type 2C/R5, or LGMD2C/R5 — in children with this form of LGMD in the U.S.
What Is Duchenne Muscular Dystrophy (DMD)?
2022年1月19日 · The DMD gene is located on the X chromosome, which is one of the two sex-determining chromosomes in people: usually, females have two X chromosomes, while males have one X chromosome and one Y chromosome. DMD is much more common in males than in females because females get two X chromosomes and therefore copies of the DMD gene — so if one copy is mutated, the other copy is usually able to ...
Muscular Dystrophy News Home | Muscular Dystrophy News
2024年11月25日 · 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 …
Amondys 45 (Casimersen) - Muscular Dystrophy News
2023年10月18日 · How does Amondys 45 work? DMD is a rare genetic disease wherein mutations in the DMD gene lead to a complete or near complete lack of functional dystrophin, a protein essential for muscle health. Over time, people with DMD have symptoms of progressive muscle weakness and degeneration.. The largest in the human genome, the DMD gene consists of 79 exons, which are portions of a gene that code ...
What Is Limb-Girdle Muscular Dystrophy (LGMD)?
2022年1月24日 · Limb-girdle muscular dystrophy, or LGMD, refers to a group of different disorders that are characterized by muscle wasting at the shoulders and the hips.The disease’s name, first coined in 1954, refers to the characteristic weakness at the “shoulder girdle” and “hip girdle” — the bony structures surrounding the shoulders and hips, respectively.
Agamree (vamorolone) for Duchenne Muscular Dustrophy (DMD)
2024年1月19日 · How does Agamree work? In Duchenne, mutations in the DMD gene cause no production of the dystrophin protein that’s important for protecting muscle cells when they contract. As a result, patients have symptoms of progressive muscle damage and wasting. Chronic inflammation contributes to the progression of this damage over time.
MD and Your Heart Monitor: 6 Things I Wish I'd Known
2018年1月23日 · 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 approves oral givinostat, as Duvyzat, to treat Duchenne MD
2024年3月22日 · Weeks before the decision was expected, however, the FDA extended its deadline by three months to allow added time for regulators to review additional information submitted by the company as part of a new drug application.. The decision from the FDA was met with excitement by the DMD community. “We are pleased that the FDA approved Duvyzat (givinostat) for individuals with Duchenne age six ...
Life Expectancy - Muscular Dystrophy News
Muscular dystrophy refers to a group of genetic muscle-wasting disorders. All muscular dystrophies cause muscle weakness, but life expectancy can vary dramatically between types of muscular dystrophy. While some cases may be mild and progress slowly, others can cause severe muscle weakness and disability.
Life Expectancy Increasing for DMD Patients, 40-year Analysis Finds
2022年2月1日 · Survival probabilities decreased with age. It was 99.8% at age 10, 59.5% at age 20, 26.1% at age 30, and 13.3% at age 40. Further analysis suggested an improvement in life expectancy over time.