At the end of June, SMA researchers and clinicians from around the world met in Orlando, Florida, for the 2023 Annual SMA Research & Clinical Care Meeting. The annual meeting provides an opportunity for SMA scientists and clinicians to share their most recent discoveries and learnings, and is an incubator for collaborations, including cross-disciplinary […]
Thank you to everyone who attended the 2023 Annual SMA Conference at Disney’s Yacht & Beach Club in Orlando, Florida! It was an impactful weekend of opportunities to connect, learn, and have fun. We are happy to announce that, including on-site registration, there were over 3,200 attendees in total – a record for the Annual
Thank You for an Amazing 2023 Annual SMA Conference! 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.
Chicago, June 30, 2023 – Cure SMA receives a surprise gift in the amount of $500,181 from the Luke 18:1 Foundation, a 501(c)3 organization founded in Dallas, TX, at the 2023 SMA Annual Conference. The Luke 18:1 Foundation was created by Daniel and Nicole Stickane in honor of their son, Luke, who was diagnosed with
Below is an excerpt from a Scholar Rock press release: Scholar Rock, a Phase 3, clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced new data from the Phase 2 TOPAZ trial extension period evaluating patient outcomes at 36 months of treatment with
