Spinal muscular atrophy

Unconventional drug ‘lottery’ will hand out 50 doses of Zolgensma, world’s most expensive gene therapy
The lottery that began [recently] was not about money, or about choosing a school, or about obtaining a visa. It ...

Viewpoint: Outrage over $2 million price tag for new gene therapy is perplexing and disappointing
As someone who has lived with spinal muscular atrophy for all 30 years of my life, I was perplexed and ...