KEI letter to Roche requesting voluntary license to patents on risdiplam, a treatment for Spinal Muscular Atrophy (SMA)
SMA
risdiplam
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
Access to SMA therapy: Iman’s Story
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
Interview with Tasnim Jadwat, regarding access to treatments for SMA
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
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
Zolgensma R&D subsidies
Charity and 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