Cure SMA’s New Year Letter for 2020

Dear SMA Community,

We are excited to be kicking off a new year and a new decade full of promise for the SMA community. Over the past week on our social channels, Cure SMA has been highlighting the past decade of progress and seeing all we have achieved has made us optimistic for what comes next. In that spirit, we wanted to share a look ahead.

In 2020, we expect to reach another major milestone in the SMA drug pipeline. In late May, the U.S. Food and Drug Administration (FDA) is expected to issue a decision on risdiplam, an oral drug that increases the amount of SMN protein in the body. With the prospect of three treatment options for individuals with SMA, we remain committed to doing everything we can to ensure all individuals and families are educated about these cutting-edge therapies so they can make the best treatment decisions and get the best the care they need. This includes continuing to advocate for broad and timely insurance coverage, increasing the number of sites that can provide these therapies, and removing administrative or other barriers to treatment.

The changing SMA landscape will require a new approach to care and support as those with SMA live longer and healthier lives. The need for more customized support programs in local communities has led to the expansion of our one-day “Summit of Strength” events in 2020, with dates and locations to be announced in the coming weeks. The Cure SMA Care Center Network will seek to have 24 committed partners across the country by the end of the year, bringing us closer to the goal of creating an evidence-based standard of care that will improve the lives of all those affected by SMA. And finally, the ongoing evolution of our support programs for all ages and stages of SMA will include revisions to the current Care Series Booklets, as well as new resources to focus on the social impact of SMA.

As we continue to expand our support programs, we also remain focused on the future of research. In 2020, we will continue to fund basic research to “seed” the SMA drug pipeline with new research ideas beyond survival motor neuron (SMN) protein. Many researchers believe we will have the most impact by combining SMN treatments with therapies that address new biological systems and processes, like muscles and the neuromuscular circuitry. Our goal is that all those affected by SMA will have access to a combination of therapies that can be targeted to their individual goals and needs. We also continue our work to expand the number of sites participating in SMA clinical trials, so that all SMA clinical trials can proceed as quickly and efficiently as possible.

This year will also see continued adoption and implementation of newborn screening for SMA. Already, 13 states have fully implemented newborn screening for SMA, with another six states screening newborns for SMA through pilot projects. By the end of the year, we expect that approximately two-thirds of the states in the U.S. will have adopted newborn screening for SMA.

Finally, we will continue our investment in support for individuals and families living with SMA, and the Annual SMA Conference remains the cornerstone of our community engagement initiatives. We look forward to seeing many of you at the 2020 Annual SMA Conference taking place June 11-14, 2020, as we head to Disney’s Yacht & Beach Club Resort in Orlando, Fla.

The new year is a great time to set up to reach big goals. In 2020, we expect to meet two important benchmarks for the community—where more than 50 percent of all newborns in the U.S. are screened for SMA, and where more than 50 percent of all currently affected individuals with SMA are being treated with an approved therapy. We cannot do that without your unwavering support and partnership.

Thank you for all you do, and I look forward to keeping you updated on all our efforts throughout the coming year.

Very best,
Kenneth Hobby
President, Cure SMA

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