2 Million Dollar Baby

A family with a sick baby was raising money for their baby who was very ill and needed treatment. The babies’ name is Lucy Van Doormaal and was born with a spinal muscular atrophy which is a genetic disease that causes infants’ muscles to waste away, killing them before the age of 2. The treatment for this is a one-time gene therapy. This family spent months raising money for this treatment which is $2.125 million dollars. 


Although this family had raised 2 million dollars for their babies’ treatment, they were given the treatment for free and got to keep all the money they saved for their babies’ treatment. They received this treatment for free because of a program for sicknesses like that. It is called Manage Access Program which is a program from the drug company, Novartis Gene Therapies which selects a child in need of it which gives them the treatment for free which luckily Lucy had been chosen. 


Because of this program Lucy got a one- time infusion in her vein to cure her illness. SMA is an inherited disorder stemming from a defective gene that leads to the death of nerve cells that causes the movement of muscles that allows people to walk, talk, breathe, and even swallow. This is what slowing causes them to die before the age of 2. Unfortunately for Lucy, she had 1 SMA which is the more severe and aggressive type of disease as a baby. It would cause her to barely be able to crawl, talk, or breathe. 


Lucy was given the treatment just a few days after her second birthday. Some say it was a miracle for her to even make it past or close to the age of 2 without the treatment. After she had the treatment, her parents were able to notice changes in her. She was able to hold her head up by herself which she had never done before they say. They are hoping that soon she will be able to crawl and walk.  As for Lucy she is lucky to see changes, this is because the treatment is not a guaranteed cure. 


The treatment is approved in the U.S. and in other countries except for Canada which is where this family is located. However, the British Columbia Children’s Hospital was able to provide Lucy with this special treatment. Because of this family receiving this treatment, other countries who did not before allow this treatment are getting the program for children who do not have access to this kind of help and are giving them the therapy they need. 


This program has allowed 100 children under the age of 2 to have access to therapy and even the treatment. As for Lucy they didn’t know until weeks after she was born. When Lucy was born, they said she was sucking her thumb right away but after a few weeks, they noticed her shortness of breath and her not being able to move her thumb up to her mouth. After they noticed this they took her to the hospital and got the terrible news of her illness. Until they were given the treatment for free and are really happy to have her healthy and safe once again.