News Related to Homozygous Familial Hypercholesterolemia

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LIB Therapeutics launches Lerochol in the United States after FDA approval

May 11, 2026

LIB Therapeutics launched Lerochol in the United States after FDA approval for lowering LDL-C in adults with hypercholesterolemia, including HeFH. The once-monthly PCSK9 inhibitor is available with a $199-per-month cash-pay option.

Ultragenyx Reports Q4 2025 Results, Resubmits UX111 Gene Therapy Application to FDA

Feb 13, 2026

Ultragenyx reported Q4 2025 revenues of $207 million, up 25% year-over-year, while announcing a 10% workforce reduction and resubmission of its UX111 gene therapy application following positive long-term clinical data.