Imagine that your child reaches their developmental milestones as expected for the first six to 18-months of their life. Then, without any warning, stops progressing and begins to lose the communication and motor skills they’ve just started to learn.
When most families find out their child has Rett syndrome, it’s the first time they’ve ever heard of this rare and complex neurodevelopmental disease. Rett syndrome affects approximately 6,000-9,000 people in the U.S and causes severe impairment that affects nearly every aspect of a patient’s life.1-2
There is no cure, and until this spring there was no therapy specifically approved for the treatment of Rett syndrome.3
For the first time during October’s Rett Syndrome Awareness Month, families have renewed hope. Texas parent Kassie Morrell speaks about the 24/7 care that both of her identical twin daughters Kenzie and Kaylie (10 years old) need and the multidisciplinary approach their care team has taken for nearly a decade to manage the girls’ significant motor, behavior and communication challenges that impact every facet of their daily lives. Kassie will also address why the FDA approval of the first ever treatment for Rett syndrome is so important to her, Kenzie and Kaylie, and the entire Rett syndrome community.
Joining her is clinical geneticist Dr. Steven A. Skinner, Director of the Greenwood Genetic Center in Greenwood, S.C., who can describe Rett syndrome, the challenges patients and families face as it progresses, and discuss the latest in research and treatment.
Interview is courtesy: Acadia Pharmaceuticals
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