Genentech, a member of the Roche Group, recently announced that the U.S. Food and Drug Administration (FDA) has granted priority review of a supplemental new drug application (sNDA) for the use of Evrysdi® (risdiplam) to treat pre-symptomatic babies under two months of age with spinal muscular atrophy (SMA). The sNDA submission incorporates interim data from the […]
We’ve recently released an update to the SMA drug pipeline. This latest version includes: 20 active programs, including three approved therapies. 10 pharmaceutical partners. 12 programs in clinical trials. An ever-increasing breadth of potential treatment approaches to SMA. Effective Treatments for All Ages, Stages and Types of SMA The updated version of this pipeline reflects
Cure SMA Releases Updated Drug Pipeline Read More »
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
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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
Every August, the spinal muscular atrophy (SMA) community unites, raises awareness, and shows our pride for SMA Awareness Month. This year, Shaakira Thomas and her family chose to go above and beyond by organizing landmark lightings all over the country, all in support Cure SMA’s newborn screening initiatives. Shaakira and her husband, Parris Sr., are parents to Aaliyah,
Community Spotlight: Shaakira Thomas and Family Read More »
