Family members of people with SMA play an active role in managing the daily lives of their loved one – from physical tasks like feeding, bathing, dressing, and more, to mentally-taxing activities like arranging paid caregivers, appointments. and treatments. What impact does this have on the family member? Cure SMA launched an online survey on […]
Survey Alert: SMA Family Caregivers Read More »
Dear SMA Community, We are excited to announce the design of SAPPHIRE, a Phase 3 clinical trial to further evaluate the safety and efficacy of apitegromab in those living with Spinal Muscular Atrophy (SMA). The SAPPHIRE study is part of Scholar Rock’s ongoing development program for apitegromab. In addition to this statement, Scholar Rock is
Cure SMA’s Jacqueline Glascock, Director of Research Programs, recently talked with Managed Healthcare Executive about information on the three FDA-approved SMA treatments and the need for combination therapy. Check out the interview here! Importance of Combination Therapy New developments in treatments for spinal muscular atrophy (SMA) over the last few years have changed what is possible
Cure SMA Discusses FDA-Approved SMA Treatments & Dissects Combination Therapy Read More »
We are pleased to share a community update on Novartis Gene Therapies’ Clinical Trial Program.
Community Update from Novartis Gene Therapies Read More »
We are pleased to share an update on Genentech’s, a member of the Roche Group, initiation of MANATEE, a new global Phase 2/3 clinical study that aims to evaluate the safety and efficacy of GYM329 (RO7204239), an investigational anti-myostatin antibody targeting muscle growth in combination with risdiplam, in Spinal Muscular Atrophy (SMA). Why a combination
Update on Genentech/Roche Initiation of MANATEE Clinical Study Read More »
This week, new data was presented at the World Muscle Society 2021 Virtual Congress. Check it out below! Scholar Rock presents two posters featuring additional analyses from the TOPAZ phase 2 trial of Apitegromab: Poster: Apitegromab in Spinal Muscular Atrophy (SMA): An Analysis of Multiple Efficacy Endpoints in the TOPAZ Trial (LBP.10) This
New Data Presented at World Muscle Society 2021 Virtual Congress Read More »
Biogen is committed to improving health outcomes for people impacted by spinal muscular atrophy (SMA) and generating data to assess if there are treatment pathways to address potential unmet needs. Today, we are excited to announce our intent to initiate a new global SMA clinical trial called ASCEND to understand whether an investigational higher dose
The advent of three approved SMN-upregulating therapeutics for spinal muscular atrophy has dramatically changed the natural history of the disease. There are, however, many uncertainties regarding timing of treatment initiation, response to treatment, and long-term outcomes. The identification of biomarkers, medical indicators that can inform us as to disease status, treatment response, and/or provide information
Cure SMA Convenes Working Group to Discuss Biomarkers for SMA Read More »
Cure SMA would like to thank everyone who attended the recent 2021 Virtual SMA Conference and 2021 Virtual SMA Research & Clinical Care Meeting. It was an impactful week of workshops, networking, and SMA community engagement. This year’s virtual conference week—the second virtual conference during these pandemic times—hosted nearly 2,500 registrants from over 65 countries. Cure SMA offered
Thank You for Another Fantastic Virtual SMA Conference! Read More »
When you first learned that you or your child had SMA, you probably began analyzing your home life, your or your child’s education, and other immediate concerns. In time, you realized that the needs would continue to evolve. How would you pay for medical expenses that insurance did not cover? What types of programs would
Financial Planning with a Disability Read More »
