Genzyme and Isis have announced the FDA approval of Kynamro (mipomersen sodium) injection indicated for homozygous familial hypercholesterolemia (HoFH). The regulatory agency approved Kynamro 200mg subcutaneous injection per week as an ...
REGENX BioSciences has received FDA orphan drug designation for treatment of familial hypercholesterolemia (HoFH) using NAV rAAV8 vectors. The REGENX program consists of a NAV rAAV8 vector expressing a normal copy of the human ...
Tags: orphan drug designation, familial hypercholesterolemia, clinical study