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 »
Scholar Rock, a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today highlighted key accomplishments of the past year and announced R&D priorities for 2020. Below please find a community statement from Scholar Rock specifically discussing SRK-015. ______________________________________________________________________________ Dear SMA Community, We at Scholar
Community Statement from Scholar Rock Regarding SRK-015 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 »
Kara and Ryan Forcier, of Stillwater, Minn., were on a rollercoaster almost immediately after the birth of their son, Zachary. Zachary was born with skin lesions that turned into blood clots, which almost led to having his limbs and digits amputated. Thankfully this was not the case, and after the skin lesions healed, Kara and
Community Spotlight: The Forcier Family Read More »
John and Krista Scurria, of Baton Rouge, La., believe in a parent’s intuition. Krista first became worried about the development of her oldest child, Josh, while she was still pregnant with him. But doctors disregarded her concern, attributing it to first-time parent naiveté. However, she and John continued to worry after Josh was born, and
Community Spotlight: Scurria Family 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 »
