Regina Philipps faced the challenges of a rare, devastating muscular disorder when her son Shane was diagnosed with spinal muscular atrophy (SMA) Type 2 at 10 months old. Each year, one in every 11,000 babies is born with the disease. SMA is the leading genetic cause of infant death. SMA progresses quickly, meaning the earliest possible diagnosis and treatment is crucial. If left untreated in one of its most severe forms, Type 1, children often require permanent feeding and breathing support or pass away by their second birthday.
When Shane was six months old, his parents saw their seemingly healthy baby boy suddenly regress in his development. While Shane had once been able to hold his head up, roll over and push himself up, his strength started to slowly disappear and he began losing his ability to move his legs and lift his arms to his mouth.
While on average, babies with SMA Type 2 are diagnosed between six and 24 months of age, Shane received his diagnosis at 10 months old. Shane was treated with another SMA treatment before he received his one-time gene therapy treatment with Zolgensma® (onasemnogene abeparvovec-xioi). Zolgensma addresses the genetic root cause of SMA by replacing the function of missing or faulty genes with a new working copy of the gene in order to restore the production of the needed protein. Zolgensma is approved in the U.S. to treat pediatric patients less than 2 years of age with SMA. Please continue reading for Indication and Important Safety Information, and please see accompanying
