Restrictions on $2 Million Drug Highlight Challenge for Gene Therapies

Ciji Green kisses her daughter, Maisie Forrest, 19 months, at her home in Grand Junction, Colorado. Maisie has spinal muscular atrophy, a progressive disease tied to the lack of a crucial gene. Zolgensma, the world’s most expensive drug, could help her survive. The family was fundraising and fighting for access. Recently, their insurance company agreed to Zolgensma only after her family appealed.

See more here, from The Wall Street Journal.