Genentech, a member of the Roche Group, today announced positive topline results from the pivotal Part 2 of the FIREFISH study, evaluating risdiplam in infants aged 1-7 months with Type 1 spinal muscular atrophy (SMA). The primary outcome measure of the study was the proportion of infants sitting without support for at least five seconds […]
The 2020 Annual SMA Conference registration numbers are growing quickly! We expect the 2020 conference – which includes both the Family Conference and the Research & Clinical Care Meeting – will be one of our biggest ever. Hotel rooms in our block are filling up, so be sure to register and make your hotel reservations
Annual SMA Conference Registration Continues to Grow! Read More »
On Tuesday, February 4th, at 11am CST (9am PST/10am MST/12pm EST) Biogen and Cure SMA will co-host a webinar for the SMA community. During the webinar, you’ll hear from Biogen about the latest information on its SPINRAZA clinical trial, DEVOTE, a Phase 2/3 randomized, controlled dose-escalating study with infantile and later-onset SMA of all ages,
Cure SMA and Biogen to Co-Host Webinar on DEVOTE Clinical Trial – Register Today! Read More »
Dear SMA Community, We are excited to be kicking off a new year and a new decade full of promise for the SMA community. Over the past week on our social channels, Cure SMA has been highlighting the past decade of progress and seeing all we have achieved has made us optimistic for what comes
Cure SMA’s New Year Letter for 2020 Read More »
During our last fiscal year—July 1, 2018, to June 30, 2019—Cure SMA funded more than $7 million in new research and ongoing research. The resources will help accelerate research and ensure we are developing treatments for all types, ages, and stages of SMA. The areas funded include: Basic research, with a focus on funding approaches
A Look Back at 2019: Cure SMA Research Year-in-Review Read More »
With an incidence of approximately one in 11,000, more than 360 infants will be born annually with spinal muscular atrophy (SMA). Newborn screening can help to pre-empt irreversible motor neuron loss, increase prompt intervention, and eliminate long diagnostic delays. The impact of early diagnosis has been demonstrated through several studies that reinforce the importance of
Dear Members of the SMA Community, As part of Biogen’s ongoing commitment to individuals living with, and caregivers affected by, spinal muscular atrophy (SMA), we are pleased to announce updates to our SPINRAZA® (nusinersen) clinical development program – an extensive clinical data set in the treatment of SMA to date that includes more than 300
Biogen Issues Q4 2019 Community Statement on Spinraza Read More »
The 9th Annual “Hope on the Hill” Congressional Dinner was held on Tuesday, December 3, 2019 at The Willard Hotel in Washington, DC. More than 180 people from the SMA community – including families, government officials, and industry partners – attended the dinner, which raised $195,000 to fund advocacy efforts, research, and family support services.
The Fall 2019 issue of Compass is now available online. In this issue we provide a one-year update on the roll-out of the Cure SMA Care Center Network and its efforts to put processes in place that will allow for the development of an evidence-based standard of care to improve the lives of people with
Fall 2019 Issue of Compass Now Available Read More »
Maria Eleni Kaloidis is a 22-year-old in her final year at Post University. Like most women her age, she enjoys spending time with her family and friends while “drinking way too much coffee,” she adds. Despite showing symptoms the first few weeks of life, Maria was not diagnosed with spinal muscular atrophy (SMA) Type 1
Community Spotlight: Maria Kaloidis Read More »
