News

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases know that the impacts of such conditions extend beyond just medical…

Oral Puldysa (idebenone), Santhera Pharmaceuticals‘ investigational treatment for breathing problems caused by Duchenne muscular dystrophy (DMD), will be available to eligible patients in the U.K. for another year via the renewal of its Early Access to Medicines Scheme (EAMS), the company announced. In agreeing to this renewal,…

Sarepta Therapeutics has submitted a full application to the U.S. Food and Drug Administration (FDA) seeking approval of casimersen (SRP-4045) to treat Duchenne muscular dystrophy (DMD) patients amenable to exon 45 skipping. “The completion of our casimersen submission is an important milestone in our journey to advance treatments…

The COVID-19 pandemic has forced many to shelter at home, caused millions of layoffs, and tightened budgets. But for families affected by Duchenne muscular dystrophy (DMD), who front extra medical costs and must decide between working and potentially exposing their son to the virus, the pandemic…

The Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency, has recommended a label update for Translarna (ataluren) to enable its use in Duchenne muscular dystrophy (DMD) patients who lost their ability to walk. The committee has specifically voted to remove…

A Phase 1 clinical trial of BBP-418, a potential first oral therapy for limb-girdle muscular dystrophy type 2i (LGMD2i), has dosed its first healthy volunteer, ML Bio Solutions announced. The company also announced that children and adults with a confirmed LGMD2i genetic diagnosis are invited to join a natural history…

A partnership between Muscular Dystrophy Canada (MDC) and Technology for Living (TFL) will seek to provide a cough assist device to any adult in British Columbia who has neuromuscular disease and needs one. Under the new partnership, adults who are registered with the…

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support activities that align with the foundation’s Rare Belonging focus, a set of funding priorities aimed at improving the…

At the one year mark, Sarepta Therapeutics’ SRP-9001 micro-dystrophin gene therapy continues to show positive results in four boys with Duchenne muscular dystrophy (DMD) enrolled in a Phase 1/2 study. The therapy improved the boys’ motor abilities and…

People with neuromuscular disorders such as muscular dystrophy are not at a higher risk of acquiring COVID-19, but they do face greater challenges should they become infected. Creating tailored plans to manage risk and adhering to them is especially important. This is the broad take-home message of a…