For many years, the annual Cure SMA Conference has been bringing the SMA community together to learn and to connect. Throughout its history, the conference has traveled to a variety of locations across the U.S., hosted countless seminars and socials, navigated new virtual platforms, and provided…
This year marked the second entirely virtual annual Cure SMA conference. Instead of armies of wheelchair users invading Disneyland and hotels, we fellowshipped through virtual trivia nights and Zoom socials. While I yearn to be back in person with my fellow SMA peeps,…
Children with spinal muscular atrophy (SMA) who started taking Spinraza (nusinsersen) before symptoms appeared retained their ability to swallow after approximately four years, while youngsters with later-onset SMA walked farther with less fatigue. These results, from analyses performed on multiple ongoing clinical trials, were presented by Biogen,…
Meaningful further improvements in motor skills were evident with apitegromab treatment in later-onset spinal muscular atrophy (SMA) patients on maintenance doses of Spinraza (nusinersen), updated Phase 2 TOPAZ trial findings show. “The TOPAZ results show that apitegromab has promising potential to benefit the large portion of individuals with SMA who still…
Evrysdi (risdiplam) continues to be linked to motor function improvements in a broad range of people with spinal muscular atrophy (SMA), according to recent data from two ongoing Phase 2 clinical trials. These data were presented at the 2021 Virtual SMA Research & Clinical Care Meeting. Presymptomatic…
