Over a 6-year period, patients with the genetic disease lipoprotein lipase deficiency (LPLD) who received a single gene therapy treatment of alipogene tiparvovec had a marked reduction in the severity ...
CATALINA FOOTHILLS, Ariz. (KGUN) — After 37 years and against all odds, one woman with lysosomal acid lipase deficiency (LAL-D) found hope and healing through a medication called Kanuma. The ...