Clinical Programs

Delivering transformational therapies for rare diseases

Our goal is to invest in science that leads to access to therapies for patients with rare diseases.

lipodystrophy patients

In This Section

Currently at Novelion Therapeutics, and our subsidiary Aegerion Pharmaceuticals, we are focused on executing a robust development plan for metreleptin in a number of potential indications.

We are also developing a synthetic retinoid product candidate, zuretinol acetate, for the potential treatment of inherited retinal disease (IRD) caused by underlying mutations in RPE65 or LRAT genes, that prevent adequate functioning of the retinoid cycle. The disease areas being investigated comprise leber congenital amaurosis (LCA) and retinitis pigmentosa (RP).

For more information about any of Novelion Therapeutics' or Aegerion Pharmaceuticals' enrolling or ongoing clinical trials, please visit www.clinicaltrials.gov.