Dossier
homozygous familial hypercholesterolemia
Coverage of homozygous familial hypercholesterolemia in the Nexus archive.
- 2 Philadelphia labs receive millions in federal funding to create treatments for rare genetic diseases in 5 years
Two Philadelphia-based labs received federal funding to develop gene therapies for rare genetic diseases, aiming for human trials in three years. The funding supports research at Children’s Hospital of Philadelphia and GEMMABio, focusing on scalable treatments for Maple syrup urine disease and homozygous familial hypercholesterolemia.