Author Archives: Magdalena Kegel

A global Phase 3 trial of givinostat — an investigational treatment aiming to boost the muscle regeneration capacity in Duchenne muscular dystrophy patients — is recruiting participants across North America and Europe. The trial is looking to recruit boys, older than six years, who are still ambulatory and on a stable…

Seven pharmaceutical companies are banding together to sponsor Project Hercules — an initiative that addresses evidence generation for Duchenne muscular dystrophy (DMD) treatments. The project, led by the non-profit organization Duchenne UK, aims to simplify the way evidence is generated for health technology assessments by authorities such as Great Britain’s National…

Researchers have found that Duchenne muscular dystrophy patients usually have simultaneous neurological disorders or abnormalities, including epilepsy. Particularly, those who had epilepsy also had the neurodevelopmental condition attention deficit hyperactivity disorder (ADHD), or the neuropsychiatric conditions obsessive-compulsive disorder, anxiety or sleep disorders. Since the research, published in the European…

Researchers have succeeded in growing working muscle tissue from a skin sample — a feat that until recently was viewed as science fiction. Although the lab-grown muscle was not as strong as those in the body, the advance opens up for new approaches to tissue regeneration in patients with muscular dystrophy…

Capricor Therapeutics has received a green light from the U.S. Food and Drug Administration to launch a Phase 2 study of CAP-1002 cell therapy in children and adults with advanced Duchenne muscular dystrophy. Capricor expects to open the trial to patient enrollment in early 2018 and, if successful,…

A one-time infusion of Capricor Therapeutics’ heart progenitor cell therapy CAP-1002 improved heart muscle function and reduced tissue scarring in a small group of patients with Duchenne muscular dystrophy. The impact of the therapy, however, went beyond heart improvements, as the study demonstrated that CAP-1002 also improved hand and…

A new method using DNA isolated from a blood sample was far more sensitive in detecting large mutations causing Duchenne muscular dystrophy than currently used genetic diagnostics tools, according to a new study. The next-generation mapping method, using the Bionano Genomics platform, has the potential to replace less sensitive…

Mice with Duchenne muscular dystrophy compensate for the weakness in their breathing muscles by sending stronger signals to the nerves controlling the diaphragm, according to scientists at the University College Cork in Ireland. Now it is important to examine whether the same mechanisms exist in human Duchenne patients, researchers…

With one exon-skipping therapy — Exondys 51 (eteplirsen) — approved for Duchenne muscular dystrophy (DMD) and others in clinical trials, it’s easy to envision Sarepta Therapeutics as the “exon-skipping company.” But the idea of current exon-skipping treatments as the ultimate for DMD couldn’t be further from what Sarepta…

Parents of boys with Duchenne muscular dystrophy (DMD) as well as patient advocacy groups are praising the recent approval of Sarepta Therapeutics‘ Exondys 51 (eteplirsen) to treat the disease. But the decision by the U.S. Food and Drug Administration (FDA) to approve a treatment without clear data supporting…