Khrystal K. Davis

CEO and Patient Advocate

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Khrystal joined the rare disease community in 2011 when her newborn son, Hunter, was diagnosed with Spinal Muscular Atrophy (SMA) Type 1, the leading genetic cause of mortality in children under the age of two. SMA Type 1, often de- scribed as ALS in babies, robs the ability to move, swallow, and ultimately breathe. Khrystal is the author of Hunt for a Cure: An Unexpected Adventure to Save a Life. Khrystal founded Zebra Leaf Publishing to provide the rare disease community a platform to promote rare disease awareness.
In May of 2016, Khrystal advocated alongside the FAST Movement (Families for the Acceleration of Spinal Muscular Atrophy Treatments) in a meeting with top FDA representatives for access to Spinraza, an SMA treatment in clinical trials at the time. Together with other FAST members, she asked the FDA to stop placebo tri- als, provide a means of access for the weakest SMA patients, accelerate the ap- proval, and approve the treatment for all SMA patients regardless of age or type of SMA. In an interim look completed on August 1, 2016, the FDA found the treat- ment met trial objectives. An Expanded Access Program for SMA Type 1 patients commenced August 12, 2016, and the FDA approved Spinraza for children and adults with SMA on December 23, 2016.
Khrystal is committed to improving health outcomes in those with rare diseases through improved access to rare disease treatments. She advocates for the expan- sion of newborn screening programs and insurance policies that conform to FDA labels for orphan drugs. She is a proponent of patient-driven access to rare disease treatments.