Shaping the Future Together
Now, more than ever, we stand ready to shape a future that will change lives, and give new hope to all those who are affected by SMA. Please consider giving generously to help shape our future, together.
101% of $750,000.00 Goal
Raised: $ 762702 Goal: $ 750000
Thanks to the dedication of our families, the generosity of our supporters, and the ingenuity of our research partners, we have reached a new phase in our mission to develop a treatment and cure for SMA.
On December 23, the FDA announced that it has approved Spinraza for SMA, making it the first-ever approved therapy for SMA. This is an incredible milestone for our community.
What’s more, five other drugs are currently in clinical trials, and one of those, AveXis’s gene therapy program, has received breakthrough designation from the FDA: another first for SMA.
Shaping the Future of Treatment
Because of the progress in our drug pipeline, we’ve launched a number of urgent initiatives to give drug programs the best chance of success. As part of our patient-focused drug development initiative, we’re working with the FDA to ensure our community’s voice is taken into account when treatments come forward for approval. We’re advocating for these drugs to receive the broadest label possible. And we’re at the forefront of insurance issues, so cost is not a barrier to potentially life-change therapies, ensuring the broadest access to treatment possible.
An FDA-approved treatment will change the course of SMA, but that first approval is not the end of our path. SMA is a complex disease, affecting many different systems, pathways, and processes in the body. To treat all ages, stages, and types of SMA, we’ll need new treatments that address these other areas, working in combination with therapies that address the underlying genetics of SMA.
Shaping the Future of Care
As FDA-approved treatments extend the lives of those with SMA, care will become more and more important. Our goal is not only to extend life, but also to ensure the best possible quality of life.
We’re currently working to develop a network of clinical care centers that will collaboratively collect patient data to answer questions and develop strategies for optimal clinical management of SMA. With this project, we hope to bring improved care for SMA to as many families as possible.
For More Information
For more information on how Cure SMA is working shape the future of treatment and care for our community, please see these recent updates from our news section.
- Cure SMA Participates in a Special FDA Listening Session
- SMA Community Conducts Outreach Visits with Members of Congress
- 6th Annual Congressional Dinner Furthers SMA Advocacy Work
- SMA Community is Granted a Patient Focused Drug Development Meeting with the FDA
- Cure SMA Launches Insurance Coverage and Payment Policy Project
- Cure SMA Joins SMA Europe and TREAT-NMD for Meeting with EMA Regulators
- Cure SMA Announces Newborn Screening Initiative in Partnership with MDA
- Cure SMA Announces $2.5 Million in New Planned Research Funding
- Cure SMA Posts Keynote Presentations on the NDA Process
- 2016 Update on SMA-FDA Interactions
- Cure SMA Launches New Surveys to Share Our Community’s Experience
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