Aug. 10, 2015
Posted by Lisa Sencen
Cure SMA is a voluntary-driven, non-profit organization dedicated solely to eradicating SMA by promoting and supporting research, helping families cope through informational programs and support, and educating the public and the medical community about SMA.
1.) What does it mean to you personally to be a patient organization serving the rare community?
Spinal muscular atrophy is the number one genetic cause of death for infants. But even so, like many rare diseases, SMA is not always well known in the wider population. Families affected by SMA may at first feel alone or unsure of what to do next. CureSMA is proud to be one of the first places that families can turn in this situation. We hear from approximately 70% of all newly diagnosed families each year. We provide care packages, informational packets, medical equipment, and more. We offer scholarships to our Annual SMA Conference, and a special program for newly diagnosed families. And we have 33 chapters around the US, so these families can connect with other SMA families in their area. We also offer these same resources to families who are currently living with SMA.
In addition to being able to offer reliable, unbiased information, and a caring community, we are proud to be able to share with these families that there is real hope. Cure SMA is the leader in advancing research to develop therapies with almost $60 million raised and funded so far. While much work still remains to be done, we have tangible reasons to believe that we can reach our ultimate goal of a treatment and cure for SMA.
2.) What do you find your patient community values most from your organization?
Our community values the dedication, expertise and resources that we bring to research for spinal muscular atrophy. Our success in advancing research through our comprehensive research model has allowed us to leverage relationships into the pharmaceutical community, and we now have seven clinical trials in progress. Because of this, SMA is now close to an approved treatment. Our community also values the objective evaluation we bring to research, and the careful balance we make between hope and hype
But perhaps even more than all of this, our community values the compassion, inspiration, and passion that we all bring together—whether at an event, in local chapters, or at our Annual SMA Conference.
3.) What are some of the challenges your organization has faced?
The SMA drug pipeline is now both broad and deep, as we know that, statistically speaking, the chance of any one drug being approved is small. We have reasons for great optimism when we look at the full picture, but we must remain cautious about the success of any one program. Maintaining this balance can be challenge, particularly as we move closer to an approved treatment. And as an approved treatment gets closer, it often seems that frustration increases.
It’s likely that the first treatment we get will be one that slows or stops disease progression, rather than an outright cure. Because of this, we’ll need more medical professionals and more resources for patient care, as individuals live longer but still deal with the effects of SMA.
4.) Whats been your most successful awareness campaign and/or fundraising event?
Each year, we host 300+ fundraising and awareness events, all across the US through our chapters. These local efforts are by far our most successful and most meaningful events in any given year. They vary from lemonade stands to formal galas. The dedication of our event organizers is what drives these to be successful, and the power of the community coming together is what makes them meaningful.
925 Busse Road
Elk Grove Village, IL 60007
NORD is excited to be putting the spotlight on CureSMA this week! Stay tuned for posts about CureSMA on NORD social media throughout the week. If you would like your NORD Member Organization to be in the spotlight for the week, email your NORD Membership Manager for more information.