On Feb. 25, the US Food and Drug Administration (FDA) granted accelerated approval to casimersen (Amondys 45) for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 45. It is the fourth exon-skipping, disease-modifying drug to treat DMD, the most common childhood form of muscular dystrophy. The drug is administered by intravenous . . .
The urgency of the COVID-19 pandemic has transformed the drug-development landscape, driving rapid development, US review, and, in one case, new therapeutic approval. To bring critical drugs to market, the US Food and Drug Administration (FDA) is utilizing a special emergency program announced in April 2020 to accelerate approval of promising coronavirus therapies, an initiative . . .
Researchers at the University of Pittsburgh invite parents and guardians of children with Duchenne muscular dystrophy (DMD) to participate in a study that investigates whether they utilize mental health resources and if they have encountered any barriers to accessing such resources. For this study, researchers at the University of Pittsburgh will ask participants to take a . . .
In 1996, MDA provided a small starter grant to Dr. Steve Wilton, a primary investigator at the University of Western Australia in Perth, for research into what scientists then thought was a radical process to address the effects of certain gene mutations. A quarter of a century later, this process — exon skipping — has . . .
Researchers at the University of Rochester in New York seek people diagnosed with Duchenne muscular dystrophy (DMD) who are interested in helping to develop disease-specific patient-reported outcome measures for future clinical trials. This survey will help to determine the most critical symptoms of children, young adults, and adults with DMD, and as a result, it . . .
September is Newborn Screening Awareness Month, and today, Sept. 7, is World Duchenne Awareness Day, dedicated to spreading knowledge about Duchenne muscular dystrophy. As we honor both, we’re providing an update on our work to ensure every state in the US screens babies at birth for DMD, leading to improved outcomes and longer, stronger lives. . . .
On Aug. 25, Sarepta Therapeutics announced that the US Food and Drug Administration (FDA) accepted the company’s New Drug Application (NDA) seeking accelerated approval for casimersen (SRP-4045), an investigational therapy for Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping. The FDA has granted Sarepta’s application Priority Review Status, meaning that a decision is expected . . .
Today, the US Food and Drug Administration (FDA) granted accelerated approval to viltolarsen (Viltepso) for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. It is the third exon-skipping, disease-modifying drug to treat DMD, the most common childhood form of muscular dystrophy. The drug is administered by intravenous infusion. Viltepso will . . .
On June 15, Sarepta Therapeutics announced positive results from its phase 1/2a study of SRP-9001 gene therapy to treat Duchenne muscular dystrophy (DMD). Data from four patients indicated that a single intravenous infusion of SRP-9001 was safe and well tolerated, with no serious adverse events. Additionally, all participants demonstrated improvements across multiple efficacy-related endpoints, including . . .
In April, MDA fielded a survey to ask its community how COVID-19 was impacting their lives. We heard your responses — anxiety, questions, hope — and wanted to know more. In this six-part blog series, Hacking COVID, people from MDA’s community, all living with neuromuscular diseases, shared how they’ve altered their day-to-day lives, how they’ve . . .