My name is Wade Hosey! I will be 2 on November 21, 2019. I was just diagnosed with Spinal Muscular Atrophy. SMA is a disease that robs people of physical strength by affecting the motor nerve cells in the spinal cord, taking away the ability to walk, eat, or breathe. It is the number one genetic cause of death for infants. There is a new treatment that can only be given before I turn 2 in just four months. This drug has a high success rate of stopping the progression of SMA, however, it is also the most expensive drug in the world being 2.1 million dollars for the 1 time injection.
Spinal Muscular Atrophy (SMA) is a genetic disease passed down to the child when both parents are carriers. 1 in 50 people are carriers and 1 in 10,000 babies are affected. If a child is born with SMA the gene called ‘Survival of Motor Neuron’ is either missing or mutated. This gene produces a protein. If this protein isn’t produced in sufﬁcient amounts, motor neurons die. Motor neurons are nerve cells in the spinal cord that send out nerve ﬁbers to muscles throughout the body and control their movement.
Without the production of this protein, the muscles used for movement become weak and deteriorate.
Since being diagnosed with SMA, we have become more aware of some of the effects on my little body, such as tremors and my muscle weakness. The good news is SMA does not affect my ability to think, learn, and build relationships with those around me. I am very smart and I am beginning to talk. I am the sweetest, happiest baby! I am friendly and love interacting with people around me. I seem to have a keen sense of empathy and notice when others around me have walking difﬁculties and other physical problems. I watch with intent, concern and love. When I am around my friends and cousins and they are running and playing, I try very hard to ﬁt in. I am so thankful for my cousins who love me just like I am!
There is a new life-saving therapy that costs $2.1 million called Zolgensma. This is a treatment for children with Spinal Muscular Atrophy. The treatment delivers a gene that sits beside the bad/missing gene and delivers the protein that is needed to help my muscles stay strong. There have been trials indicating a high success rate.
Zolgensma is delivered using the AAV9 virus. Most people build an immunity to this virus as they grow. Because I do not have any antibodies against this virus, I am still a candidate for this treatment. We must act quickly to secure the funding.
This drug is very expensive but it could save my life! I am very grateful for your help no matter how big or little you can share. Currently, my insurance does not cover this treatment.
Thank you for your prayers, love and concern.