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Alagille Syndrome
Conference Roundup

Long-term maralixibat treatment improves height in children with ALGS

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Long-term treatment with maralixibat significantly increased height from baseline to week 204 in children with Alagille syndrome (ALGS), according to a poster presented at NASPGHAN 2022.

Data from patients who participated in the maralixibat clinical studies and the long-term open-label extensions were used to evaluate height and weight z-scores.

The greatest improvements in height and weight z-scores were found in patients who had the lowest scores at baseline. At week 48, patients that had the greatest catch-up weight gain also had the greatest catch-up height growth.

A greater accelerated height was also noted in patients who achieved a serum bile acid threshold <200 µmol/L. The authors suggest that this may mean bile acid homeostasis may facilitate improvement in height deficits.

Reference
Maralixibat Improves Growth in Patients with Alagille Syndrome: A 4-Year Analysis. Presented at: NASPGHAN 2022.

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