Celebrating Skylar's 21st Birthday with 21 Miles - 14 Miles Done, 7 Miles to Go
Check out this video to see how.
Families of SMA (now Cure SMA) was founded in 1984. The SMN gene was discovered one year later. This discovery provided researchers with an understanding of how to target and treat Spinal Muscular Atrophy (SMA).
Skylar was diagnosed with SMA Type 1 and given a prognosis of 2 years. At the time, his pediatrician had never heard of the disease. Families of SMA, not restricted by HIPPA laws in 2002, reached out to us to provide support, resources, and hope. There was only one treatment in development.
Skylar's Aunt Maggie launched the inaugural Skylar's Fight swim as part of Sharkfest San Francisco (aka Escape from Alcatraz) to raise awareness and funds for Families of SMA. Our first team consisted of 11 swimmers.
The number of swimmers, supporters, and donations for Skylar's Fight continued to increase over the next two events. Families of SMA focused their research on RNA-based therapies and preclinical use of small molecules (RXs). Skylar participated in a study that helped determine the different types of SMA (I, II, III, and IV) based on the number of copies of the backup gene SMN2 he carried.
Skylar became an angel, losing his fight with SMA. Donations were made to Families of SMA in his memory. Six drugs moved into clinical development.
Skylar's Fight was rebranded to Reach4Sky. Our biennial event moved to Southern California as part of Sharkfest San Diego in 2011, 2013, 2015, and 2017, and the Oceanside Pier Swim in 2019. Families of SMA became Cure SMA in October 2014 as research moved into high gear culminating in three FDA-approved treatments and 20+ in development, including 1) Spinraza, the first-ever treatment for all types of SMA, FDA-approved in December 2016, 2) Zolgensma, a gene replacement therapy (through a one-time infusion) for infants under two, FDA-approved in May 2019, and 3) Evrysdi, a daily oral medication, FDA-approved In August 2020. While these treatments are changing the landscape of SMA, they are not a Cure. We will continue our work until we can extinguish SMA!