News

The U.S. Food and Drug Administration decided not to approve Translarna (ataluren), by PTC Therapeutics, as a treatment for specific types of Duchenne muscular dystrophy caused by “nonsense” mutations. Although not entirely unexpected, the decision was disappointing to many in the Duchenne community. On Sept. 28, an FDA advisory…

Respiratory activity is significantly impaired in Duchenne muscular dystrophy (DMD), but a new mouse study suggests that enhancing breathing via the brain may improve respiratory capacity. The study “Sensorimotor control of breathing in the mdx mouse model of Duchenne muscular dystrophy” was published in The Journal of Physiology. Respiratory failure…

Researchers have found that reducing levels of elevated sarcolipin protein may be a potentially effective therapeutic strategy in the treatment of Duchenne muscular dystrophy (DMD) patients. The study, “Reducing sarcolipin expression mitigates Duchenne muscular dystrophy and associated cardiomyopathy in mice,” was published in Nature Communications. Current therapies…

More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars are being invested in clinical studies that will test new ways of evaluating — and advancing — potential treatments, including the use of natural history…

Casey’s General Stores  customers contributed $1.35 million in August to support the Muscular Dystrophy Association‘s 2017 Summer Camp, where children living with MD enjoy a week of fun and make new friends at no cost to their families. Since the fundraising campaign was launched in 2006, $9 million has been…

Two non-profits that work on standards that can facilitate drug development have created a guide to help those doing Duchenne’s muscular dystrophy research. The two are Critical Path Institute (C-Path) and the Clinical Data Interchange Standards Consortium (CDISC). The resource they created is the Duchenne Muscular Dystrophy Therapeutic Area User…

As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives it offers pharmaceutical companies to develop therapies for rare diseases — off the table. That’s the message being pushed by the National Organization for…

Tricyclo-DNA (tcDNA), a class of therapeutics known as antisense oligonucleotides (AONs), has shown an important degree of effectiveness and a promising safety profile in a preclinical study with mice. The goal of the study is to test tcDNA’s potential use in treating Duchenne Muscular Dystrophy (DMD). The study, “Efficacy and…

The newly approved Duchenne muscular dystrophy therapy Exondys 51 (eteplirsen) stays in immune cells several days after leaving the blood, continuing to help regenerate muscle fibers, according to a study in mice. Researchers at the Children’s National Health System in the Washington area said the findings on immune cells known as…

The biotechnology company Capricor Therapeutics recently presented data that support the use of its peripheral intravenous delivery method in the upcoming HOPE-2 clinical trial testing the investigational agent CAP-1002 as a potential therapy for Duchenne muscular dystrophy (DMD). The data were presented during a poster session at the Alliance…