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 . . .
There’s no doubt 2020 has been a challenging year, but the commitment and passion of our MDA advocates have truly been a bright spot. As we close out the year, let’s recap your amazing accomplishments. Access to care and therapies from day one Thanks to the actions of advocates, together we made sure the lifesaving . . .
On Oct. 27, Scholar Rock announced positive results from an interim analysis of its phase 2 clinical trial evaluating SRK-015, a therapy for treating spinal muscular atrophy (SMA). After six months of treatment with SRK-015, patients with SMA types 2 and 3 demonstrated significant motor function improvements, as measured by the Hammersmith scale, with higher dose . . .
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. 7, the US Food and Drug Administration (FDA) granted approval of risdiplam (Evrysdi) for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. It is the third disease-modifying therapy approved to treat SMA, the leading genetic cause of infant death. The drug is an orally administered liquid, the first medicine that could . . .
Josh Cueter, 22, from Troy, Michigan, is a 2020 graduate from Michigan State University. At school he was a board member of the MSU Adaptive Sports & Recreation Club as well as MSU’s Council of Students with Disabilities. After a semester internship with the Equal Employment Opportunity Commission in Washington, DC, Josh is currently working . . .
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 . . .
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 . . .
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 . . .
On Jan. 22, Genentech, a member of the Roche Group, announced positive results from the second part of its pivotal phase 2/3 FIREFISH clinical trial assessing risdiplam in infants age 1 to 7 months with spinal muscular atrophy (SMA) type 1. The company announced that the study met its primary endpoint, which was a proportion of infants . . .
