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The FDA approved tofersen for amyotrophic lateral sclerosis based on the drug’s ability to lower blood levels of neurofilament light (NfL) — establishing a proof of potential for this neuroscience biomarker that could have implications for other diseases of the brain.
Joni Rutter, director of the National Center for Advancing Translational Sciences (NCATS), discusses the centre’s audacious goal to have 25% of diseases covered by the experimental drug pipeline by 2032.
The last decade has seen major progress in the molecular diagnosis of rare inherited diseases, underpinning gene-targeted therapies that are now in the clinic. However, new treatment development is slow and very expensive. Here, we highlight the opportunities for mitochondrial disorders, the challenges and some of the potential solutions.
