3 ms·
A cure for muscular dystrophy for humans already exists. Only problem is, it costs a whopping $2 million USD. [1] https://www.reuters.com/article/us-novartis-g
by gzer0 5y ago
A cure for muscular dystrophy for humans already exists. Only problem is, it costs a whopping $2 million USD.
[1] https://www.reuters.com/article/us-novartis-genetherapy/novartis-2-million-gene-therapy-for-rare-disorder-is-worlds-most-expensive-drug-idUSKCN1SU1ZP https://www.reuters.com/article/us-novartis-genetherapy/nova...
[2] https://www.npr.org/sections/health-shots/2019/05/24/725404168/at-2-125-million-new-gene-therapy-is-the-most-expensive-drug-ever https://www.npr.org/sections/health-shots/2019/05/24/7254041...
- Dylan16807 5y agoAlso you can only use it on babies. But as pointed out elsewhere, wrong disease. As for that drug, I doubt the price would need to be that high if people were ordering a thousand doses a year, but I'm no expert.
- fullburley 5y agoThat's for one type of muscular dystrophy, SMA, there are many other types without great treatment options. Lifelong exon skipping treatments recently approved for subsets of DMD are >1M USD per year, and result in dystrophin expression at only a few percent of "normal" (see data from trials of casimersin).
- staticautomatic 5y agoMuscular Dystrophy isn’t a single disease— it’s an umbrella term for a whole class of neurodegenerative diseases with different etiologies. We need to be clear on which one we’re talking about when we talk about cures.
- ben_bai 5y agoThe industry is going the route of gene therapy. > SAREPTA THERAPEUTICS ANNOUNCES GENE THERAPY PROGRAM FOR LGMD2A/R1 Today https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-executes-licensing-agreement-gene-therapy https://investorrelations.sarepta.com/news-releases/news-rel...
- gpt5 5y agoInteresting. Hopefully the insane price will actually do good by incentivizing more gene therapy innovation.