New Zolgensma data demonstrate age-appropriate development when used early, real-world benefit in older children, and durability 5+ years post-treatment Novartis Gene Therapies recently shared new data that reinforce the transformational benefit of Zolgensma ...
Dear Members of the SMA Community, Each year, the entire rare disease community comes together on the last day of February to recognize and raise awareness for the 300 million ...
Genentech, a member of the Roche Group, announced in a press release that Evrysdi™ (risdiplam) data from the dose finding Part 1 of the pivotal FIREFISH study in infants with symptomatic Type 1 ...
New developments in treatments for spinal muscular atrophy (SMA) over the last few years have changed what is possible for people with SMA, allowing them to achieve new developmental milestones. However, these treatment ...
Below we have provided an update from Biogen regarding the status of its DEVOTE clinical trial. For more information about SPINRAZA (nusinersen), visit https://www.curesma.org/spinraza/. ++++++++++++++++++++ Biogen's DEVOTE study is designed to evaluate the ...
Scholar Rock, a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced 6-month interim results from their clinical program for its ...
Genentech, a member of the Roche Group, today announced new 2-year data from Part 1 of the pivotal FIREFISH study of Evrysdi™ (risdiplam) in infants aged 2-7 months with symptomatic Type 1 spinal ...
Dear SMA Community, We recently received important feedback from the U.S. Food and Drug Administration (FDA) following their review of data from the STRONG study of AVXS-101 intrathecal (IT) formulation in older patients ...
Biogen Inc., today announced, it plans to initiate a global Phase 4 clinical study, RESPOND, to examine the clinical benefit and assess the safety of SPINRAZA (nusinersen) in infants and children with spinal muscular atrophy ...
In the midst of the ongoing Coronavirus (COVID-19) global pandemic, Cure SMA is excited to announce its new funding for the Pediatric Neuromuscular Clinical Research Network (PNCRN). In 2020, Cure SMA will provide ...
Cure SMA is proud to share with the SMA community that it has published a paper in the Orphanet Journal of Rare Diseases, titled ‘The SMA Clinical Trial Readiness Program: Creation ...
New SPINRAZA® (nusinersen) Data Reinforce Sustained Efficacy and Longer-Term Safety Across Broad Range of SMA Patients Biogen Inc. recently announced additional data from the SPINRAZA (nusinersen) clinical development program that further demonstrate the ...
