14. The story of oligonucleotides and spinal muscular atrophy

In this episode, Conor tells Dodi the story of Roy Muhrbeck. It all started 4 years ago when new parents, Hugo Muhrbeck and Maja Ödmann brought home their first child. They were excited and nervous as so many first time parents are. But after a short while, they started to think that maybe everything wasn’t exactly how it should be. Soon after, their son Roy was diagnosed with spinal muscular atrophy (SMA). 

Stay tuned to find out how oligonucleotides (oligos) help Roy and where he is today. 

Transcript

Keywords: spinal muscular atrophy, SMA, antisense oligo therapy, genetic disease, Ionis Pharmaceuticals, genomics, clinical trial, oligonucleotides.