The FDA has approved the first drug to treat the devastating genetic disease Spinal Muscular Atrophy.
This is the greatest Christmas gift my family could have received. My granddaughter Scarlet was born with the condition.
SMA is caused by a defective gene that inhibits the body's ability to create a protein necessary for muscle development.
The drug was originally called Nusinersen. It is now labeled as Spinraza.
It is designed to stimulate back-up genes to create that missing protein.
In trials, 3-year-old Jace Dorer of Royal Oak has shown considerable improvement on the drug.
This news means Scarlet will most likely be able to gain access to the drug early in 2017.
