Written by Martha Harlam
Until 1986 I led a wonderful life. I got to sing for my supper in musicals and opera. Now, spinocerebellar ataxia was taking control of my movements. The hereditary neurological disease was making theater directors declare me unfit and a danger to others on stage. Stage podiums were becoming more technical and required total bodily control, but my balance was off. My singing career was over, and my life became one of depression, totally consumed with my disability.
As the progressive nature of the disease continued, so did my search for a purpose in my life. My loss of gainful employment led to being an ataxia patient advocate. I started a website, Miracle Answer, to help help spread awareness and help others to understand ataxia as a disease.
A new purpose in life was rekindled in me. I had lost all hope and belief that research could find, in my lifetime, a solution for people with certain neurological disorders. There is no cure for Ataxia, but the families of children with neurological conditions and children with rare undiagnosed diseases are hoping that gene therapy will soon be available to help them.
With that mission in mind, the Global Gene Research Initiative was developed. The Global Gene Research Initiative (GGRI) gives new hope to children and young people affected by genetic neurological disorders and the rare disease community. Now, at age 72, I have found a new passion to advocate for: gene therapy.
Alongside advocacy, biotech, pharmaceutical manufacturers, researchers at children’s hospitals, logistics, charities globally can all come together under one roof. We are all pinning our hopes on the future of gene therapy!
Editor’s Note: Get Involved
Thank you to Martha for stepping forward to share his story of spinocerebellar ataxia hope and advocacy. At Patient Worthy, we know rare diseases and chronic conditions doesn’t discriminate. Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have a rare disease journey to share, reach out here to learn more about how your voice can help spread awareness and inspire individuals from all walks of life.
