Researchers at Mitsubishi Tanabe Pharma Development America, Inc. (MTPA) are seeking adults living with amyotrophic lateral sclerosis (ALS) to participate in a phase 3b clinical trial to evaluate the safety and efficacy of oral edaravone to treat ALS. Edaravone (brand name Radicava) was designed to help protect cells from damage caused by free radicals, and was . . .
On September 24, Capricor Therapeutics announced positive results at the World Muscle Society Virtual Congress from its phase 2 HOPE-2 trial of the investigational therapy CAP-1002 for treatment of people in advanced stages of Duchenne muscular dystrophy (DMD). The primary and secondary endpoints of the study, the improvement of upper limb and cardiac function, were met, . . .
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 . . .
2021 is a new year: The country has a new administration, and a new Congress has been sworn in. What isn’t new is MDA’s commitment to working tirelessly in Washington, DC, to transform the lives of people living with neuromuscular diseases (NMDs). Here’s a sneak peek at some of the issues we’ll be working on . . .
We know families across the country, especially those in the neuromuscular disease (NMD) community, are concerned about the spread of coronavirus and its associated disease, COVID-19, and the impact on their loved ones. In these uncertain times, MDA’s advocacy team will not stop serving and amplifying the voices of those in the NMD community, especially . . .