Cure SMA Featured in Assay and Drug Development Technologies

Cure SMA was featured in the July/August issue of Assay and Drug Development Technologies, a scientific journal that focuses on the development of new drugs. In honor of SMA Awareness Month, SMA was also featured on the front cover.

Assay reaches scientists in 170 countries worldwide, making this a unique opportunity for us to influence SMA research. Especially during August, we work to spread awareness of SMA among our friends, family, and co-workers. In the same way, we’re also working to spread awareness of SMA throughout the scientific community.

The issue featured a how-to article on preclinical drug development for SMA, including how to discover, validate, and optimize drugs that enhance survival motor neuron (SMN) protein levels. Individuals with SMA do not produce this protein at high enough levels.

The lead author for this article is Jill Jarecki, PhD, Cure SMA’s research director. The article’s other authors are an interdisciplinary group from academia, industry—including Pfizer and PTC Therapeutics—and SMA advocacy groups.

The issue also featured an interview with Jill on Cure SMA’s research funding strategy, and the progress that we’ve made in finding a treatment and cure for SMA.

Our thanks to the rest of the paper’s authors—Drs. Jonathan Cherry, Dione Kobayashi, Maureen Lynes, Nikolai Naryshkin, F.D. Tiziano, Phillip Zaworski, and Lee Rubin—and to the editor-in-chief of Assay, Dr. James Inglese of the National Center for Advancing Translational Sciences (NCATS) at NIH. Dr. Inglese is also a member of the Cure SMA Translational Advisory Council.

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