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Single drug provides first evidence of 'nearly universal' pharmacological chaperone for rare disease

A study published in Nature Structural & Molecular Biology is the first time researchers have shown evidence that a single drug, already licensed for medical use, can stabilize nearly all mutated versions of a human protein, regardless of where the mutation is in the sequence.

from Medical Xpress - latest medical and health news stories https://ift.tt/YXl0s6D