On November 1, 2024, Knowledge Ecology International (KEI) submitted a proposal for the addition of risdiplam to the World Health Organization’s (WHO) Model List of Essential Medicine (EML). You can access the full proposal here: risdiplam2WHOEML1Nov2024 The EML, updated every… Continue Reading →
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 →
KEI has a page on Charity and NIH funding related to Zolgensma, here: https://www.keionline.org/charity-nih-funding-related-to-zolgensma
June 14, 2019 Zolgensma, the Novartis brand name for the new $2.1 million gene therapy for treatment of Spinal Muscular Atrophy (SMA), appears to be a remarkable medical breakthrough treatment. It is also, like all of the new cell- and… Continue Reading →
