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A group of Croatian patients with dwarfism and multiple hormone deficiencies (GH, TSH, PRL, FSH/LH) related to homozygous PROP1 mutations do not experience diabetes mellitus, early grey hair, or death (40)

Nature Genetics, 47 (1), 73-77

Third, gene therapy approaches face unresolved safety concerns, including off-target mutagenesis, immune responses against Cas9, and potential oncogenesis from integrating viral vectors [139]

The pain is severe, and patients often cannot wear socks or touch bedsheets during flare-ups

You might feel a little discomfort where the IV is, but our team ensures you stay comfortable