Great Progress, Greater Need

In the last few years, we’ve gotten closer to an approved treatment than we ever have been. Through research funded by Cure SMA, we’ve identified the cause of SMA, and are developing multiple ways to treat SMA.

But with this great progress comes even greater needs. That’s why we’ve set a goal of raising $750,000 by December 31, as part of our 2016 Annual Campaign. Your year-end gift will be crucial in helping us meet these greater needs.

Great progress, greater demand.

Thanks to our community’s awareness efforts, more newly diagnosed families are reaching out to us. Our clinical care research and medical professional education mean that many of those affected by SMA have better access to improved care.

This means a greater demand for Cure SMA’s family support and patient care services. More families are coming to our Annual SMA Conference, requesting our care packages or info packets, or needing our equipment pool. And when new SMA drugs are approved, care and support will be even more critical.

We need to increase our funding for patient care and family support, to serve a growing community with new and more complex concerns.

Great progress, greater opportunity.

As research progresses, we are focusing on ensuring we have treatments for all types, all ages, and all stages of SMA. Several treatments that address the underlying genetics of SMA have reached clinical trials. Researchers are also developing drugs that can treat SMA in other ways—for example, by addressing the issues that impact the muscles and nerves.

We need to increase our funding for research to build upon the current drugs in development, and ensure we have effective treatments for all in our community.

Great progress, greater urgency.

Seven SMA drugs are now in clinical trials—and several are getting closer to potential FDA approval. With the immense promise of new treatments comes a new challenge: navigating the clinical trial and regulatory issues for a new drug approval.

We need to increase our funding for clinical trial and regulatory initiatives, to act with urgency in gaining an approved treatment for SMA.

We need to increase our efforts to support clinical trials. We also need to make sure our community’s voice is heard, so the FDA can make the best decisions on approvals when that time comes.

Please give today. Your generosity has brought us this great progress. And with your support, we will continue to meet the needs of our community.

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