Genentech/Roche has provided the below community statement with clinical trial updates for SUNFISH (Type 2/3). The first patient is now enrolled in SUNFISH, as the trial advances to part two, which is designed to be a pivotal marketing registration trial. Dear SMA Community, We are pleased to share an update on the SUNFISH study evaluating […]
Genentech/Roche Releases Clinical Trial Update for SUNFISH (RG7916) Read More »
A recording of last week’s webinar updating the community on Spinraza access and administration sites is now available online. A PDF of the webinar presentation is also available for download. An update on the current status of dosing in states across the US included the following information: As of September around 2,000 patients had been dosed, with
Recording of the September Webinar on Spinraza Updates Now Available Read More »
AveXis, Inc., a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the U.S. Food and Drug Administration (FDA) has notified the company that based on review of data submitted, including the potency assay, it may initiate its planned pivotal trial of AVXS-101 for patients with
To celebrate the FDA approval of Spinraza, Cure SMA contributed an article to the September special issue of the journal Gene Therapy. The issue , “Spinraza and Advanced Therapies: a stakeholder special,” covers a variety of perspectives and includes comments and review papers from SMA research funders, people affected by SMA, experts in the advanced
Cure SMA Contributes Article to a Special Issue of the Journal Gene Therapy Read More »
At our Annual SMA Conference in July, we announced a grassroots campaign to implement newborn screening for SMA in all 50 states. We know that newborn screening and early treatment is the best chance we have to change the course of SMA for the next generation and beyond. We challenged our community to use August,
Newborn Screening Advocacy Update Read More »
Novartis has released the following update for the LMI070 (branaplam) clinical trial, on behalf of the program team. Dear SMA community, For years, our researchers have been working hard to develop a new treatment for SMA. One promising compound in our neuroscience pipeline is LMI070, which started development for the treatment of SMA a few
Novartis Releases Update on LMI070 (Branaplam) Clinical Trial Read More »
Over the past fiscal year—from July 1, 2016, to June 30, 2017—Cure SMA has funded over $2.5 million in new research funding. This funding will be used strategically to 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 particular
Cure SMA Announces $5.05 Million in Research Funding for FY18 Read More »
Cure SMA is expanding its role in spinal muscular atrophy medical affairs at the national level through its Medical Advisory Council (MAC). The MAC sets the agenda for proactive, creative, and collaborative leadership on issues that improve the quality of medical care for those affected by SMA. The focus of the Council is to build
Applications for Cure SMA Medical Advisory Council Due October 15th Read More »
Genentech/Roche recently provided the following community statement with clinical trial updates for SUNFISH (Type 2/3). The SUNFISH trial is advancing to part two, which is designed to be a pivotal, marketing registration trial. Dear SMA Community, We are focused on progressing our studies with our investigational molecule RG7916 with utmost speed and quality and are
Genentech/Roche Releases Clinical Trial Update for RG7916 Read More »
The SMA community celebrated an impactful and advocacy-driven Awareness Month through a number of ways this August. Whether hosting candlelight vigils, meeting with state and local legislatures, holding fundraisers, coordinating MLB awareness games, and more, the community worked together to raise the general publics awareness of SMA. Advocacy Visits On August 2nd, members of the
2017 SMA Awareness Month Recap Read More »
