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Lipid storage myopathy, metabolic abnormalities resembling MADD, and deficiencies in numerous respiratory chain enzymes are the hallmarks of this disorder, which is treatable

Surface-engineered smart nanocarrier-based inhalation formulations for targeted lung cancer chemotherapy: a review of current practices

Finally, combining vectors with biocompatible scaffolds or hydrogels may extend intra-articular retention and promote localized expression

This may lead to high levels building up in the blood
