Oral treatment with AdipoRon reduced inflammation in muscle, promoted muscle fiber proliferation, and improved muscle and motor function in a mouse model of Duchenne muscular dystrophy (DMD), a study showed. Anti-inflammatory effects were also seen in muscle cells from people with DMD. The scientists said AdipoRon has the potential…
This week, I celebrate my one-year anniversary of writing columns for Bionews Services. A lot has happened in that year: I graduated from college, interned at The Washington Post, and secured a full-time newswriting position at Bionews. I’ve laughed a…
Treatment with Vyondys 53 (golodirsen) for 48 weeks increases levels of dystrophin protein by 16-fold in boys with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping, updated results of a Phase 1/2 trial show. Also, the newly produced protein was correctly located in the membrane of muscle…
The Muscular Dystrophy Association (MDA) is launching MDA Let’s Play, a platform to raise funding and awareness about muscular dystrophy and related neuromuscular disorders through gaming. “We’ve always been an iconic American charity with innovative fundraising programs to achieve our mission of transforming lives — from the telethon to…
March 1 was my one-year anniversary with Bionews Services, the publisher of Muscular Dystrophy News. Defining a period in this manner allows me to see how far I’ve come in the past year. My columns have focused on a number of topics. In my column from May 2019, I…
Treatment for six months with Increlex (mecasermin), an injectable form of insulin-like growth factor-I (IGF-I), enhanced growth in boys with Duchenne muscular dystrophy (DMD), a Phase 1/2 trial shows. However, the hormone therapy had no effects on muscle function. The study, “Recombinant human insulin‐like growth factor‐1…
The sixth annual Napa in Newport wine auction raised more than $1 million to support CureDuchenne’s mission to extend and enhance the lives of people with Duchenne muscular dystrophy (DMD) and find a cure for the disorder, the nonprofit group said. Held Feb. 29 at the…
The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…
On Saturday morning, Feb. 22, I awoke with excited anticipation of the day ahead. That day, I attended the Los Angeles Abilities Expo for the second year in a row. Attending the 2020 LA Abilities Expo was a different experience this year simply because I…
A new clinical trial is seeking about 80 people with limb-girdle muscular dystrophy type 2i (LGMD2i) to gather information on this disease’s progression that may help in advancing potential therapies and in establishing a biomarker. The observational study (NCT04202627) is now enrolling patients, ages 10 to 65, at…
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