Imagine learning your child was diagnosed spinal muscular atrophy (SMA), the leading genetic cause of death in infants. SMA causes the loss of motor neurons and progressive muscle wasting. Now imagine there is a drug that could not only save your child's life, but also improve her quality of life. The drug, Zolgensma, is the potential answer to your prayers, but it is only available for children under two-years-old. Your daughter is less than one month from her second birthday and every waking moment of your day is spent fighting for the word "approved". This is the story of my friends Rachael and Patrick Brown, and their fierce fighter, Kate. Due to Rachael and Pat's relentless advocacy on July 11, 2019, Kate received her dose of Zolgensma at Boston Children's Hospital just twelve days before her second birthday.
SMA research, including funding to develop Zolgensma, is provided by CURE SMA. In addition to funding SMA research, CURE SMA provides invaluable direct support to families battling SMA, including specialized equipment to make daily life more manageble.
It has been a year since that life saving dose, and what a year it has been. Kate is now a fiesty three-year-old, who has found her strong and funny voice, is mastering her wheelchair, and is determined to walk. She loves playing wth her brother and sister, and nothing makes her smile like making Alexa play the soundtrack of Trolls so she can dance and sing. With the support of her family, doctors, caregivers, and support from CURE SMA, Kate will continue to fight and overcome the challenges to come.
For the second year, I am running the Falmouth Road Race in August in support of CURE SMA and for Kate. This year the race will be virtual, but still a vital part of the fundraising efforts for CURE SMA. Please consider donating to support this amazing organization. Thank you!