SMA Drug Trial Goes So Well It Ends Early

This Los Angeles Times article on a new treatment for SMA Type 1. This is very promising news and I believe it will lead to additional breakthroughs for Type 2,3 and 4. SMA is different then SBMA (Kennedy's Disease), but antisense technology is being used in other progressive disorders and could be the bridge to a treatment.

Below is an excerpt from the Times article. You can read the entire article by following this link: Ionis shares leap 30% after a drug trial goes so well, it ends early

"The only option

Spinal muscular atrophy occurs in about 1 out of 6,000 to 10,000 births. It's caused by mutations in a gene that reduce production of a protein needed for survival of movement-controlling spinal neurons.

Nusinersen increases production of the protein from a closely related gene by altering how RNA made from the gene is translated into protein. This is done with antisense technology, a field pioneered by Ionis.

Drugs based on antisense technology are meant to block or change how targeted genetic instructions delivered through RNA affect the making of proteins, which are the building blocks of life. For instance, they could block certain mutations from resulting in protein synthesis that causes an unwanted medical condition.

Nusinersen has been granted orphan drug status in both the U.S. and the European Union — indicating it shows promise in treating a rare disease or condition — and it’s designated for fast-track review in the United States."

Image:  http://www.slideshare.net/pooranachithraflowry/antisense-therapy-48602109