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Muscular 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.

The 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-rel...

Also 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.

That'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).

Interesting. Hopefully the insane price will actually do good by incentivizing more gene therapy innovation.