risdiplam

Risdiplam, originally developed under contract with the SMA Foundtion by PTC therapuetics and licensed to Roche for sale under the brand name Evrysdi, is a medication used to treat spinal muscular atrophy and the first oral medication approved to treat… Continue Reading

Zolgensma

Zolgensma is a gene therapy for spinal muscular atrophy (SMA) that was approved by the US FDA in May 2019, at a price of $2.1 million per patient. Zolgensma was licensed from a children’s hospital where it was developed on… Continue Reading

UPenn, Nationwide Children’s Hospital refuse to disclose which patents they licensed to Novartis for Zolgensma

Zolgensma, Novartis’s new $2.1 million gene therapy for children with spinal muscular atrophy (SMA), is a remarkable breakthrough with a steep price tag: the cost per child is $2.125 million. The treatment was developed using research funded by the National… Continue Reading