RARE Daily

Cytokinetics and Cure SMA Renew Partnership to Advance Education and Awareness of SMA

August 26, 2020

Rare Daily Staff

Nonprofit foundation Cure SMA and biotech company Cytokinetics renewed their partnership to increase education, awareness, public policy, and fundraising for spinal muscular atrophy, a genetic disease that robs people of their physical strength and can take away their ability to walk, eat, or breathe.

SMA is a severe, progressive neuromuscular disease that can be fatal. It is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein. This protein is found throughout the body and is essential to the function of nerves that control muscles and movement. Without it, nerve cells cannot function correctly, leading to muscle weakness over time. Depending on the type of SMA, an individual’s physical strength and their ability to walk, eat or breathe can be significantly diminished or lost.

SMA occurs in 1 in 6,000 to 10,000 live births each year and is one of the most common potentially fatal genetic disorders. There are four types of SMA classified according to age of onset, with the infantile type 1 having the worst prognosis. With the recent introduction of SMN-directed therapies, it is expected that patients may live longer, but will still have a significant need to address ongoing disabilities related to respiration and mobility.

Cytokinetics will lend support for several of Cure SMA’s upcoming initiatives across the United States and continues as a member of the Cure SMA Industry Collaboration, a partnership between Cure SMA and pharmaceutical companies to address topics critical to advancing drug development in SMA.

Cytokinetics will sponsor two events in the coming months to help fundraise and increase awareness of SMA in the local community, participating as a sponsor of the Virtual Walk-n-Roll program where SMA families will be walking and rolling around their neighborhoods for Cure SMA, and supporting Cure SMA’s Evening of Hope: A Virtual Masquerade, an evening of inspiration, celebration, and hope uniting the SMA community across the country.

“We have seen significant progress in the treatment of patients with SMA, yet there is still a pressing need to increase awareness and access to care, especially during a time when families are facing many unknowns due to COVID-19,” said Kenneth Hobby, president of CureSMA. “We appreciate our ongoing partnership with Cytokinetics to enable our local and national initiatives.”

Photo: Kenneth Hobby, president of CureSMA



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