We also plan to begin dosing patients in our Phase 3 study of volanesorsen in patients with familial partial lipodystrophy by the end of the year.
Akcea and Isis initiated a Phase 3 study of volanesorsen in patients with familial partial lipodystrophy.
Mutations in LMNA cause familial partial lipodystrophy (FPLD), a genetic disorder that leads to unbalanced body fat distribution (Owen et al.
2001) and can cause an inherited disorder called familial partial lipodystrophy (FPLD), which is characterized by abnormal body fat distribution.
In 1998, researchers localized a human gene that caused familial partial lipodystrophy
, called Dunnigan type (FPLD).
1999), familial partial lipodystrophy
(Shackleton et al.
In people with Dunnigan-type familial partial lipodystrophy
, fat melts from arms, legs, and buttocks while depositing in the head, neck, and abdomen.
Findings from the first poster titled, "Metabolic Effects of Metreleptin Treatment in Familial Partial Lipodystrophy
(FPL)," included data from an ongoing expanded access protocol of metreleptin administration in patients with rare forms of inherited or acquired lipodystrophy.
Akcea's most advanced program, volanesorsen, is in Phase 3 development to treat patients with ultra-orphan lipid disorders that are characterized by extremely high triglycerides and ApoC-III, including familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy
NASDAQ: ISIS) and its wholly owned subsidiary, Akcea Therapeutics, today announced the start of a Phase 3 study to evaluate the efficacy and safety of volanesorsen in patients with familial partial lipodystrophy
We have numerous clinical trial initiations planned, including the Phase 3 study in cardiomyopathy being conducted by GSK and the Phase 3 study evaluating volanesorsen in patients with familial partial lipodystrophy
A second Phase 3 study of volanesorsen is planned to begin later this year in patients with familial partial lipodystrophy