“It’s almost as common as cystic fibrosis and yet the [national research] budget for CF was $130 million [in 2009], while the budget for SMA was $25 million,” Kissel said.
Just a month ago, Cleveland.com put out an article verifying the seriousness of a condition that I must admit I had never heard of until a friend told me about her cousin this afternoon. With 1 in 40 people unknowingly carrying the gene responsible, Spinal muscular atrophy is “the leading genetic cause of death in children under the age of 2” (Townsend, The Plain Dealer).
How is it so easily overlooked?
Perhaps it is because, since Type 1, the most serious and the most common, affecting 50-75% of sufferers, usually steals a child away before they can enter school or many of our daily lives, leaving parents to look back at the tragic busyness of their last two years and wonder whether anything more could have been done. Yet there’s always hope.
“Dramatic breakthroughs […] have led the disease to the brink of treatment”. (smafoundation.com)
Sophia’s Cure is #8 in the running for a 250,000 donation from Pepsi which would go entirely towards the research that is closing in on a cure.
The NIH has listed SMA as the Neurological disease closest to a cure, and as a gateway to curing other diseases. The research is so close that it could be in time to save the lives of many children […] Most funding is raised by the families affected by this disease. We would split the grant between 2 programs that can move into human clinical trials for a treatment of this disease within 2 years.
Voting ends June 30th. There’s still time (& other ways to make your vote count). Thanks!