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New Study Opens New Door for ALS Drug Discovery

Credit: National Cancer Institute

Credit: National Cancer Institute

1st-ever evidence-based description of neuronal protein clumps thought to be important in Amyotrophic Lateral Sclerosis (ALS) announced today. These clumps are toxic to the type of neurons that die in patients with ALS. This research could be a crucial step toward developing drugs to stop the creation of the clumps and stem the progression of the disease.

“This study is a big breakthrough because it sheds light on the origin of motor neuron death and could be very important for drug discovery,” Nikolay Dokholyan, PhD. Patients with ALS suffer gradual paralysis and early death as a result of the loss of motor neurons, which are crucial to moving, speaking, swallowing, and breathing.

The study focuses on a subset of ALS cases – 1 to 2% associated with variat...

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