- Phase III trials on Nusinersen, an antisense oligonucleotide drug that modifies pre-mrna splicing of SMN2 gene, improved motor functions of infant with spinal muscular dystrophy. http://www.nejm.org/doi/full/10.1056/nejmoa1702752 3 comments science
Linking pages
- The pharma industry from Paul Janssen to today: why drugs got harder to develop and what we can do about it | Alex’s blog https://atelfo.github.io/2023/12/23/biopharma-from-janssen-to-today.html 5 comments
- This may be a turning point in treating neurodegenerative diseases | Medical research | The Guardian https://www.theguardian.com/science/2017/dec/15/this-may-be-a-turning-point-in-treating-neurodegenerative-diseases 0 comments
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