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Brainina et al., 2019), developing physiologically relevant assay conditions (Lang et al., 2024), and implementing comprehensive approaches that combine multiple assessment methods

These evaluations allow for timely adjustments to dosing, supportive therapies, and recovery strategies while tracking progress throughout the treatment program

The limited approval landscape reflects the pharmaceutical industry's cautious approach to pediatric metabolic medications

Treatment of mutant lysates or supernatants with the reducing agent DTT (dithiothreitol) collapsed practically all the protein to monomeric size, suggesting that disulfide bonds, either intramolecular or intermolecular, are required for observation of multimers and larger species on denaturing sodium dodecyl sulfate polyacrylamide gel electrophoresis (SDS-PAGE)