In the RESILIENT SMA study, Biohaven announced today that taldefgrobep alpha showed clinically meaningful improvements in motor function at all time points on the Motor Function Measurement-32 scale (MFM-32), but the treatment arm did not statistically separate on the primary outcome at Week 48 compared to the placebo+standard of care (SOC) group. Biohaven plans to […]
Table of Contents What is SMA How Common is SMA Types of SMA Causes of SMA Carrier Testing Symptoms of SMA Diagnosis SMA Newborn Screening Treatment for SMA Outlook / Prognosis Living with SMA Latest Updates on SMA SMA Research SMA Resources and Programs Clinical Trials and Studies Common SMA Questions Next Steps for SMA
What is Spinal Muscular Atrophy Read More »
“There is an endearing tenderness in the love of a mother to a son that transcends all other affections of the heart,” said Washington Irvine. When watching River Rolle wriggle in his mother Dee’s lap while he sweetly places his hand on her chin and looks into her eyes, there is nothing more apparent than
Community Spotlight: Dee and River Rolle Read More »
In Sarah Manuel’s career as a school psychologist, she mentors elementary, middle and high school students as they navigate everyday challenges in life and education. Though it took time and self-acceptance, she is confident that living with SMA helps her relate to students on a deeper level. “Just because there are limits that a diagnosis
Community Spotlight: Sarah Manuel Read More »
This week, bills to address everyday challenges faced by individuals with spinal muscular atrophy (SMA) took important steps forward in the U.S. Congress. A key aviation bill that includes a section that will make air travel safer and more accessible for passengers with disabilities, including SMA, was approved by the U.S. House of Representatives by
Big Week in Washington for Cure SMA-Supported Legislation Read More »
Nieves and Valerie Gomez’s SMA story started in 2013, when their son Liam Taylor Gomez was born with SMA Type 1. Liam passed away at six months of age, and at that time Nieves made a commitment to dedicate the rest of his life to healthcare and helping others. Since then, he has worked in
Donor Spotlight: Nieves and Valerie Gomez Read More »
Cure SMA is pleased to announce the launch of the second annual State of SMA report. The purpose of this report is to share highlights from Cure SMA’s three databases: a patient-reported database with data from over 9,700 affected individuals worldwide that also incorporates longitudinal data from our annual community update survey; the SMA clinical
Cure SMA’s 2022 State of SMA Report Read More »
A record-number of individuals with spinal muscular atrophy (SMA), a neuromuscular disease, travelled to Orlando to attend Cure SMA’s national conference from June 29 to July 2. Several adults with SMA and families of children with SMA reported damage to their wheelchairs and other air travel hiccups that impacted their conference experience. These latest travel
Biogen Inc. announced new SPINRAZA® (nusinersen) data aimed at answering critical questions for the spinal muscular atrophy (SMA) community. The data were presented at the SMA Research & Clinical Care Meeting hosted by Cure SMA this week in Orlando, FL. Interim Clinical Outcomes from RESPOND RESPOND is an ongoing two-year, phase 4 open-label study to evaluate
Genentech, a member of the Roche Group, announced today new long-term data for Evrysdi® (risdiplam) from the open-label extension (n=50) of the pivotal FIREFISH study, reinforcing its sustained efficacy and safety profile in children with Type 1 spinal muscular atrophy (SMA). FIREFISH is a two-part study in babies aged 1-7 months at the time of enrollment.
