News

Final results of a Phase 1 clinical trial support the safety of losmapimod and suggest its effectiveness as a treatment for facioscapulohumeral muscular dystrophy (FSHD). The data, “Phase 1 Clinical Trial of Losmapimod in Facioscapulohumeral Muscular Dystrophy (FSHD): Safety, Tolerability and Target Engagement,” were presented by Michelle…

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The event’s sponsor, the National Organization for Rare Disorders (NORD),…

Sarepta Therapeutics has taken steps to ensure the supply of Vyondys 53 (golodirsen) and Exondys 51 (eteplirsen), both approved for the treatment of Duchenne muscular dystrophy (DMD), during the COVID-19 pandemic. Also, the company continues to work with regulatory authorities to guarantee the continuation…

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…

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) —…

NS Pharma launched an expanded access program (EAP) in the U.S. to allow certain Duchenne muscular dystrophy (DMD) patients to gain access to its investigational exon-skipping therapy viltolarsen. The program will allow physicians to prescribe viltolarsen to U.S. DMD patients amenable to exon 53 skipping who meet other eligibility criteria, at…

Machine learning could be used to help diagnose specific forms of muscular dystrophy (MD) using magnetic resonance imaging (MRI) scans, a study suggests. The study, “Accuracy of a machine learning muscle MRI-based tool for the diagnosis of muscular dystrophies,” was published in the journal…

Capricor Therapeutics is expanding the development of exosome-based technologies that are being tested as a potential way to treat Duchenne muscular dystrophy (DMD). Capricor’s lead candidate for DMD is CAP-1002, which is composed of cardiosphere-derived cells (CDCs). These are cardiac progenitor cells, meaning they are able to…

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…

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…