In the United States, HoFH, an orphan indication, occurs in approximately one in one million individuals.
FORBES: FDA Approves Mipomersen For Homozygous Familial Hypercholesterolemia
HoFH patients often develop premature and progressive atherosclerosis, a narrowing or blocking of the arteries.
FORBES: FDA Approves Lomitapide For Homozygous Familial Hypercholesterolemia
The safety and effectiveness of Kynamro were evaluated in a clinical trial of 51 patients with HoFH.
FORBES: FDA Approves Mipomersen For Homozygous Familial Hypercholesterolemia
Rader got funding from the Doris Duke Charitable Foundation to test the drug in six patients with HoFH.
FORBES: How A Mourning Entrepreneur And A Dogged Doctor Rescued A Rare Disease Drug
To restrict access to therapy with JUXTAPID to patients with a clinical or laboratory diagnosis consistent with HoFH.
FORBES: FDA Approves Lomitapide For Homozygous Familial Hypercholesterolemia
The FDA approval for KYNAMRO is supported by the largest clinical trial conducted to-date in the HoFH patient population.
FORBES: FDA Approves Mipomersen For Homozygous Familial Hypercholesterolemia
The safety and effectiveness of KYNAMRO have not been established in patients with hypercholesterolemia who do not have HoFH.
FORBES: FDA Approves Mipomersen For Homozygous Familial Hypercholesterolemia
The safety and effectiveness of JUXTAPID have not been established in patients with hypercholesterolemia who do not have HoFH.
FORBES: FDA Approves Lomitapide For Homozygous Familial Hypercholesterolemia
For those with HoFH, heart attacks and death often occur before age 30.
FORBES: FDA Approves Mipomersen For Homozygous Familial Hypercholesterolemia
To restrict access to therapy with KYNAMRO to patients with a clinical or laboratory diagnosis consistent with homozygous familial hypercholesterolemia (HoFH).
FORBES: FDA Approves Mipomersen For Homozygous Familial Hypercholesterolemia
In December 2012, the FDA approved Juxtapid (lomitapide) to reduce LDL-C, total cholesterol, apolipoprotein B, and non HDL-C in patients with HoFH.
FORBES: FDA Approves Mipomersen For Homozygous Familial Hypercholesterolemia
The vote was close, with 9 for approval and 6 against, and only for treatment in a rare genetic disease called homozygous familial hypercholesterolemia (HoFH).
The big question now is how many people actually have HoFH.
FORBES: Aegerion Cholesterol Drug Approved. Will It Treat 300 Patients, Or 3,000?
There is no doubt that patients with HoFH need new medicines.
FORBES: Aegerion Cholesterol Drug Approved. Will It Treat 300 Patients, Or 3,000?
As part of its commitment to HoFH patients, Genzyme has developed KYNAMRO CornerstoneSM, an HoFH and KYNAMRO support program for healthcare providers, patients, and their families.
FORBES: FDA Approves Mipomersen For Homozygous Familial Hypercholesterolemia
Previous estimates said there were only 400 HoFH patients in the U.S. but included only those people confirmed to have two defective copies of the LDL receptor gene.
FORBES: How A Mourning Entrepreneur And A Dogged Doctor Rescued A Rare Disease Drug
The question of how many HoFH patients there are is key not only to Aegerion, but to Isis Pharmaceuticals, which has its own HoFH drug, Kynamro, awaiting FDA approval.
FORBES: Aegerion Cholesterol Drug Approved. Will It Treat 300 Patients, Or 3,000?
The U.S. Food and Drug Administration today approved Kynamro (mipomersen sodium) injection as an addition to lipid-lowering medications and diet to treat patients with a rare type of high cholesterol called homozygous familial hypercholesterolemia (HoFH).
FORBES: FDA Approves Mipomersen For Homozygous Familial Hypercholesterolemia
Last night, the Food and Drug Administration announced the approval of Kynamro, a new drug to lower cholesterol in patients with homozygous familial hypercholesterolemia (HoFH), a rare genetic disease that causes sky-high cholesterol levels and heart attacks at a young age.
FORBES: After 24 Years Of Struggle, Has Stan Crooke Finally Birthed An Age Of Antisense?
KYNAMRO, given as a 200 mg weekly subcutaneous injection, has been approved as an adjunct to lipid-lowering medications and diet to reduce low density lipoprotein-cholesterol (LDL-C), apolipoprotein B (Apo B), total cholesterol (TC), and non-high density lipoprotein-cholesterol (non HDL-C) in patients with homozygous familial hypercholesterolemia (HoFH).
FORBES: FDA Approves Mipomersen For Homozygous Familial Hypercholesterolemia
应用推荐