Life Saving Spinal Muscular Atrophy (SMA) Treatment | Sam's Story

Spinal muscular atrophy (SMA) is a hereditary disease that causes progressive weakness and can lead to death. Before a gene therapy medication was developed that changes the way the broken gene in SMA is expressed, there was no way to slow the progression of weakness. Thanks to a huge team effort at Johns Hopkins, this life saving medication has been given over 500 times and has made a profound impact on patients’ lives. Meet the patient who received the 500th dose. #SpinalMuscularAtrophy #JohnsHopkins https://www.hopkinsmedicine.org/neuro...