They are diseases that threaten more than physical health: memories, personality, and the ability to move and speak are incrementally stolen. And until this year neurodegenerative diseases, from Alzheimer’s to ALS, had been entirely unstoppable.
However, a breakthrough in Huntington’s disease this week suggests this bleak picture could be about to change. The landmark trial was the first to show that the genetic defect that causes Huntington’s could be corrected, raising hopes that the drug will become the first to slow the progress of the disease – or even stop it.
The Huntington’s results alone would have been remarkable enough, but they come just a month after the same experimental class of drugs were revealed to help patients with a different degenerative disease, called Spinal Muscular Atrophy (SMA). Babies with the most severe form of SMA normally never develop the strength to sit up or roll over, but after four years on the drug, some of these children are starting to stand and take their first steps with a walker.
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Since September, 16 major drug companies have inked deals with the Trump administration to lower prices....
