This is a letter to Roche requesting a voluntary license to the patents on risdiplam, a treatment for Spinal Muscular Atrophy (SMA). KEI-Risdiplan-patent-license-request-8july2022 This was the August 5, 2022 response from Roche. Roche-Response-to-Risdiplam-license-request–5Aug2022 KEI wrote to Roche again on February… Continue Reading →
Risdiplam, originally developed under contract with the SMA Foundation by PTC Therapeutics 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 →
Recent years have seen several blockbuster treatments enter the market, many rooted in publicly funded foundational and pre-clinical research, and benefiting from government subsidies. In particular, we have conducted extensive research on Spinal Muscular Atrophy (SMA), which has regularly hit… Continue Reading →
Spinal muscular atrophy (SMA) is a rare neuromuscular disorder, with an estimated incidence of between one and two cases per 10,000 births worldwide. The most severe form of the disease – SMA Type I – previously had close to 95%… 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 →
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 →
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 →