Results from a clinical trial of risdiplam in a broad range of people with spinal muscular atrophy were a highlight of SMA Europe’s 2020 conference, a board member of that SMA umbrella patient advocacy group said…
Risdiplam treatment has led to “clinically meaningful” improvements — whether gains or disease stabilization — in the broadest range of spinal muscular atrophy (SMA) patients, by age and disability level, yet enrolled in a clinical trial. More than one-third of those in the second part of the SUNFISH trial…
Risdiplam safely and significantly improved or stabilized motor function in children and young adults with spinal muscular atrophy (SMA) types 2 and 3, one-year data from the second part of the SUNFISH trial show. As anticipated by risdiplam’s developers, these benefits were more pronounced in younger patients. “Risdiplam is the…
Genentech announced, without providing details, positive topline results from the pivotal second part of its FIREFISH clinical trial of risdiplam, now before the U.S. Food and Drug Administration for approval. The company said in its release that risdiplam demonstrated statistically significant and medically meaningful motor milestone improvement…
Roche has kicked off a worldwide early access program for its experimental therapy risdiplam by making it available to Europeans with the most serious forms of spinal muscular atrophy (SMA). Europeans with SMA type 1 will be able to get it immediately, Roche told SMA News…
The U.S. Food and Drug Administration (FDA) accepted Genentech‘s application requesting approval for risdiplam, its potential oral treatment for all types of spinal muscular atrophy (SMA), and gave it priority review. An agency decision is expected on or before May 24, 2020. If approved, risdiplam would become…
Risdiplam significantly improves motor function in pediatric and young adult patients with spinal muscular atrophy (SMA) types 2 and 3, according to interim data from the SUNFISH trial. “The positive outcome of this trial is an important milestone for people with type 2 or 3 SMA, too many of whom…
Risdiplam continues to show promise as a safe and effective therapy for people with spinal muscular atrophy (SMA) types 1, 2 and 3, according to the latest results from ongoing clinical trials. Data from the FIREFISH (NCT02913482), SUNFISH (NCT02908685), and JEWELFISH (NCT03032172) clinical trials were presented at the…
Treating teenagers and adults with oral risdiplam, a potential therapy for spinal muscular atrophy (SMA), led to sustained increases in blood levels of the key SMN protein missing in these patients, the lead investigator for a Phase 2 clinical trial said in an interview. Claudia A. Chiriboga, MD,…
Treatment with the experimental oral therapy risdiplam has been enabling patients with spinal muscular atrophy (SMA) to achieve key development milestones and clinically meaningful motor improvements beyond the natural history of the disease. Susan Begelman, MD, vice president, Neurology, Nonmalignant Hematology, Influenza, U.S. Medical Affairs at Genentech,…