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Other types of antisense oligonucleotides have been approved by U.S. regulators and are being used to treat the neurodegenerative disease muscular dystrophy and spinal muscular atrophy. The compound is in clinical trials for Huntington’s disease and ALS, also known as Lou Gehrig’s disease.
Source: tatoeba (12257415)
A variety of health conditions can rob people of their ability to speak, including stroke, a neuromuscular disease called amyotrophic lateral sclerosis or Lou Gehrig’s disease, and locked-in syndrome, in which individuals are completely awake and aware of their surroundings but unable to move or communicate with the outside world.
Source: tatoeba (12312861)