PEDIATRIC HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA (HOFH)

Clinical trials for PEDIATRIC HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA (HOFH) explained in plain language.

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Special access granted for lifesaving cholesterol drug in rare cases

Disease control APPROVED_FOR_MARKETING

This program provides special access to the drug evinacumab for patients with rare, severe inherited cholesterol or triglyceride disorders who have no other treatment options. It is for compassionate use, meaning it is considered on a case-by-case basis for individual patients wh…

Matched conditions: PEDIATRIC HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA (HOFH)

Sponsor: Regeneron Pharmaceuticals • Aim: Disease control

Last updated Mar 16, 2026 15:26 UTC

PEDIATRIC HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA (HOFH)

Get alerted when new PEDIATRIC HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA (HOFH) trials appear

Special access granted for lifesaving cholesterol drug in rare cases