Spinal muscular atrophy
7,000+ rare diseases remain untreatable. The genetic revolution and federal research funding offers hope for cures, but vaccine hesitancy and a lack of newborn screening pose hurdles
There are an estimated 7,000 known rare diseases affecting 30 million people; for 95% of them, there are no treatment ...
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 ...