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Inborn Errors of Metabolism

nearly all efficacy evidence comes from animal or in vitro studies Fewer than 10 small human studies exist, none tracking use beyond about 12 weeks, so long-term safety (including cancer or fertility risk) is unknown Currently sits in a regulatory gray zone, complicating legitimate sourcing and quality assurance Side Effects: Most commonly reported issues are mild and short-lived: injection site soreness or reactions, occasional nausea, headache, and mild dizziness, typically resolving within hours to days

Iyer, R., Gupta, A., Khan, A., Hiremath, S., and Lokhandwala, Y

Thirdly, and serendipitously, the apo structures also reveal the conformation of the crucial C-terminal region that is disordered in all previous apo structures