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Loss-of-function mutations in lysosomal proteins, as seen in Niemann-Pick disease type C1 and Gaucher disease, disrupt lipid and cholesterol metabolism and result in profound neurodegeneration
It can affect quality of life
DPP-4 inhibitors can be administered in patients with impaired kidney function due to the good safety and tolerability
Not only are the medications slightly different, but people are also very different, says Kershaw
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