The supply of Emflaza (deflazacort), a treatment for people with Duchenne muscular dystrophy (DMD), will not be affected by the COVID-19 pandemic, according to PTC Therapeutics, its developer. In a statement addressed to patients and health care professionals, released March 16, PTC said it…
Parent Project Muscular Dystrophy (PPMD) has awarded a total of $148,000 to researchers at Nationwide Children’s Hospital and the University of Missouri to help better understand and improve the treatment of cardiac disease in people with Duchenne muscular dystrophy (DMD). The funding falls under PPMD’s…
Extracellular RNAs found in urine may be able to serve as non-invasive biomarkers of how patients with muscular dystrophy — especially those with an adult-onset and the more common Duchenne MD form — are responding to therapy, a study reports. Urine-sample tests for these RNAs may also lessen the need…
Taking care of patients with Duchenne muscular dystrophy (DMD) can have significant effects on caregivers, leading to a lower health‐related quality of life, poor sleep quality, depression, pain, stress, sexual dysfunction, and lower self‐esteem, a review study reports. Caregiving can also have a considerable negative impact on work life…
Wave Life Sciences’ treatment candidate WVE-210201 has been granted both orphan drug designation and rare pediatric disease designation for the treatment of Duchenne muscular dystrophy (DMD) from the U.S. Food and Drug Administration. Orphan drug designation provides Wave with seven years of market exclusivity in the U.S., tax…
Cognitive behavioral therapy improved physical activity and social participation in patients with myotonic dystrophy type 1, indicating its potential as a treatment method for these patients, according to results from a clinical trial. Findings were reported in the study, “Cognitive behavioural therapy with optional graded exercise therapy in…
American researchers have developed a new way of delivering a gene editing therapy that significantly increases the time a repaired gene can generate the protein that’s missing in a disease. This means that a gene therapy does not have to be repeated as much to help a person counteract an…
A new way of transferring the dystrophin gene — the largest known human gene — into muscle stem cells may be possible using the human artificial chromosome (HAC) vector, a study reports, suggesting it as a potential approach for gene therapy in Duchenne muscular dystrophy (DMD). The study, “Reversible immortalisation…
Aberrant signaling in the IL-6 pathway, which is involved in muscle cell maturation, contributes to the severe symptoms seen in congenital myotonic dystrophy (CDM) patients, researchers in Japan report. The study, “Aberrant Myokine Signaling in Congenital Myotonic Dystrophy” published in the journal Cell Reports, also suggests that treatments aimed at regulating…
Scientists at Saint Louis University believe that an investigational therapy called SR8278 holds potential for treating Duchenne muscular dystrophy (DMD) after it was shown to increase muscle function and decrease muscle fibrosis in lab mice. The discovery follows the team’s research of the nuclear receptor REV-ERB, which regulates key processes in…
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