Until recently there was no cure or therapy available for the patients with SMA. Anika was lucky however, if we could call a baby diagnosed with SMA lucky, that she was born at the time when something could be done. Since she was 2 months old, she has been treated with Spinraza, a treatment that could slow down the progression of the disease. Unfortunately, as time goes by, Anika’s breathing and swallowing functions are weakening and more and more assistance (mechanical and personal) is required to keep her alive.
But there is hope! A new form of gene therapy, Zolgensma, gives hope that Anika could breathe independently, and regain her strength to eat and speak.
New gene therapy
SMA is a rare, genetic disease that leads to progressive muscle weakness, until the breathing stops…
The existing evidence so far supports the positive effects of the newly approved gene therapy, Zolgensma, on the muscles of the entire body, including the breathing and swallowing muscles that are getting very weak in Anika’s case. Zolgensma is a one-time treatment, which is administered through an injection. This unique treatment, which costs staggering 2.1 million $, is able to “correct” the gene and enable it to produce the protein necessary for Anika to stay alive.