Cure SMA’s New Year Letter for 2021

Dear SMA Community,

While 2020 did not pan out as we had expected, Cure SMA remains extremely optimistic about the future for our community in 2021 and beyond. Here are the reasons why:

Research and Treatment

The research Cure SMA funds continues to expand our knowledge of SMA and deliver treatment opportunities, as reflected in the progress seen with Evrysdi, Spinraza, and Zolgensma. With these three treatments now approved by the FDA, in 2021 we are advancing new research that focuses on non-SMN targeted therapies, symptom and muscle-targeting treatments, and the development of combination therapies that increase strength and enable independence for all people with SMA.

Early Identification of SMA

As of today, 7 in 10 babies born in the U.S. are screened for SMA at birth and we are on track to exceed 90 percent of all births by this time next year. This will lead to early diagnosis and then early treatment, and ultimately better outcomes. In 2021, Cure SMA will continue working with all non-screening states to implement newborn screening for SMA to ensure babies in every state have the best chance at a healthy, full life.

Patient Care

With new information from our SMA Clinical Data Registry, we are improving our understanding of the best care for SMA. By following patient outcomes and treatment progression, we will create an evidence-based standard of care. An important goal in 2021 will be the continued growth and integration of the Cure SMA Care Center Network to ensure a comprehensive registry and improved local patient care.

Advocacy for Treatment and Services

In 2021, Cure SMA will continue its efforts to educate public and private insurers on the ongoing needs of the SMA community and the importance of access to life-altering medications. Additionally, Cure SMA will look to accelerate our work on policy issues most important to adults and young adults with SMA.

Local Support

As SMA changes, so must our efforts to unite local communities through Cure SMA Chapters, support programs including Summits of Strength, and community events like Walk-n-Rolls. With a greater local presence, we can ensure access to approved treatments and care, and we can create customized programs that meet the new needs of everyone with SMA in local communities.

This new year is a time to set up to reach big goals—and that is what Cure SMA is doing. As always, we thank you for your unwavering support and partnership and 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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