Infants with spinal muscular atrophy (SMA) who are presymptomatic and given the gene therapy Zolgensma in the first six weeks of life have better motor, respiratory, and nutritional outcomes, according to a recent study from Europe. However, researchers found functional motor scores still improved significantly, albeit less dramatically,…

Newborns diagnosed with spinal muscular atrophy (SMA) who were treated with Evrysdi (risdiplam) before the onset of symptoms are continuing to reach developmental milestones such as sitting, standing, and walking independently, with many achieving them in a time frame expected of typical child development. That’s according to two-year…

Motor function is still stable or improved among people with spinal muscular atrophy (SMA) types 2 and 3 after four years of treatment with Evrysdi (risdiplam), according to updated results from the SUNFISH clinical trial. Patients and caregivers also reported continuous improvement or stabilization in the level of…

The one-time gene therapy Zolgensma (onasemnogene abeparvovec) will now be routinely accessible at low or no cost to presymptomatic infants in England who are genetically predisposed to spinal muscular atrophy (SMA). Per updated recommendations from the country’s National Institute for Health and Care Excellence (NICE), this includes…

The total medical costs associated with spinal muscular atrophy (SMA) are lower for patients diagnosed at birth via newborn screening, compared with those diagnosed after they start showing symptoms of the disease, a new study has found. These findings “demonstrate clearly that as long as the decision to reimburse…

Children and young adults with spinal muscular atrophy (SMA) types 2 and 3 continue to report improved or stabilized motor function after three years of treatment with oral Evrysdi (risdiplam) in the SUNFISH clinical trial. “The positive long-term efficacy and safety results for Evrysdi in this broad SMA population…

Unlike in Australia, the U.S., and even several EU countries, there have been no programs for newborn screening for spinal muscular atrophy (SMA) in the United Kingdom — despite compelling evidence that an early diagnosis can improve patient outcomes. Now, Oxford University in the U.K. has launched a pilot study…

The UK SMA Newborn Screening Alliance is calling on people to take action and sign a petition requesting that spinal muscular atrophy (SMA) to be added to the U.K.’s National Health System (NHS) newborn screening program and funded. U.K. citizens and residents can sign the petition, which is collecting…

Evrysdi (risdiplam) at its approved and therapeutic dose does not lead to eye damage in children or adults with spinal muscular atrophy (SMA), data from extensive ophthalmologic monitoring of patients in its clinical trials — prompted by earlier safety findings in monkeys — show. These data support the therapy’s favorable safety…

[Editor’s Note:  This is part of a series of articles about the discovery and development of Evrysdi — the newly approved disease-modifying therapy for spinal muscular atrophy and the first oral, at-home one — as well as the scope of SMA issues and treatments. Here, we focus on…