Lowering Cholesterol Early In Life Could Save Lives Aug 6, 2008
Citing the success of lowering cholesterol levels in children diagnosed with familial hypercholesterolemia, the UC San Diego team suggests that programs to lower cholesterol in the population at large from childhood on, with the ideal LDL level set at 50 mg./dl. or less (in those at highest risk), will have a long-term beneficial effect and lower the nationwide rates of coronary artery disease. (Science Daily)
Lower kids' cholesterol the old-fashioned way Jul 24, 2008
Statins have an important role to play in children with familial hypercholesterolemia, an inherited condition, which results in very high cholesterol levels beginning at birth and can lead to heart attacks at a young age. But most kids don't needs statins to ward off a future heart attack - they need a healthy diet. (Globe and Mail)
Vytorin misses primary end point in SEAS study Jul 22, 2008
There was speculation that the trial could help rehabilitate Vytorin after the Effect of Combination Ezetimibe and High-Dose Simvastatin vs Simvastatin Alone on the Atherosclerotic Process in Patients with Heterozygous Familial Hypercholesterolemia (ENHANCE) trial was published in March, but the placebo-controlled trial design, as well as the patient population, were likely to limit the clinical impact ... In that study, investigators tested the effectiveness of combined ezetimibe/simvastatin... (TheHeart.Org)
Cholesterol kids: Rx or exercise? Jul 17, 2008
The majority of children with high LDL cholesterol have familial hypercholesterolemia, a genetic disorder not related to obesity, in which LDL cholesterol cannot be removed from the blood and builds to very high levels. People with familial hypercholesterolemia (about 1 in 500) tend to develop heart disease during early to mid adulthood. (Globe and Mail)
The Vytorin Stretch Jul 13, 2008
ENHANCE followed 720 high-risk patients with a disease called familial hypercholesterolemia for two years. CASHMERE followed 400 post-menopausal women at relatively low risk of heart disease for one year. (Forbes -- Business)
Drug Data Take Sword to Schering-Plough Apr 1, 2008
Moreover, it is questionable that a study done in patients with familial hypercholesterolemia can be generalized to the larger population. " Damien Conover, an analyst at Monringstar, says Monday's action reflected all too well what happens when money-minded investors cut to the chase after taking in medically minded information. "In my opinion it looks like an overreaction," he says, noting that a preliminary version of the data came out in January and accounted for a significant slide in share... (SmartMoney)
New data fails to quiet cholesterol drug debate Mar 31, 2008
Although the combination drug did a far better job at lowering cholesterol than Zocor alone, neither drug helped reduce the thickness of artery walls in patients with a condition known as heterozygous familial hypercholesterolemia, which causes very high levels of LDL.. Given the lack of evidence of a benefit, the ACC panel recommended that doctors first put patients on a high dose statin, and then try other drugs before reaching for Vytorin or Zetia. (Scientific American)
Vytorin On Trial Mar 23, 2008
Mevacor and other statins were developed from genetic insights gleaned from patients with familial hypercholesterolemia. In 1994 and 1995, studies of Merck's Zocor, a Mevacor replacement, and Bristol-Myers Squibb's (nyse: - - ) new Pravachol showed a striking 30% reduction in the risk of death for patients with established heart disease. (Forbes -- Business)
Story ideas from the Journal of Lipid Research Feb 8, 2008
PCSK9 degrades LDL receptors that remove LDL cholesterol (LDL-C) from the blood; mutations that result in extra PCSK9 activity produce familial hypercholesterolemia, while mutations causing less PCSK9 activity lower LDL-C and cardiovascular risk. Studies in mice have indicated that statins may increase PCSK9 expression, so Robert Konrad and colleagues studied the effect of atorvastatin (Lipitor) or placebo on PCSK9 levels in humans. (EurekAlert!)
Zetia Does Not Reduce Carotid Plaque Size When Added to Zocor Jan 16, 2008
All 720 participants suffered from an unusual condition known as heterozygous familial hypercholesterolemia. Vytorin cut "LDL levels by 58 percent, compared to a 41 percent reduction with simvastatin alone. However, the researchers found that "the average thickness of the carotid artery plaque increased by 0. (PR Newswire)
Study: Generic as effective as cholesterol drug Jan 16, 2008
The patients in the two-year study had a condition called heterozygous familial hypercholesterolemia, which the companies said affects less than 1 percent of the population. Don't Miss. (CNN -- Health)
What's Next For Vytorin? Jan 16, 2008
The ENHANCE study was conducted in 720 patients with heterozygous familial hypercholesterolemia, a genetic disease that can boost untreated cholesterol levels above 300. That high level of cholesterol makes them more likely to have heart attacks, and it should make it easier to cut down on the buildup of artery plaque. (Forbes -- Business)
Research finds Vytorin no better than generic drug Jan 15, 2008
The 720 subjects all suffered from a genetic disorder called heterozygous familial hypercholesterolemia, which affects about 0. 2% of the population. (Los Angeles Times)
Genzyme and Isis Announce Strategic Alliance Including Exclusive Worldwide License of Mipomersen Jan 8, 2008
Mipomersen's initial indication will be for patients with familial hypercholesterolemia (FH), with an anticipated filing in 2009 ... About Familial Hypercholesterolemia (FH) FH patients have high blood concentrations of LDL-cholesterol due to a genetic disorder which prevents proper metabolism of LDL-cholesterol ... Familial hypercholesterolemia can be present in two forms: homozygous (hoFH), where the same defective gene is inherited from both parents, or heterozygous (heFH), where the... (PR Newswire)
* Taiwan News Quick Take Jan 8, 2008
The boy has familial hypercholesterolemia, a rare genetic disease that blocks the circulation of cholesterol within the blood vessels. Without a transplant, he would have been unlikely to survive past the age of 20. (Taipei Times, Taiwan -- World)
Isis brings Genzyme onboard as partner Jan 8, 2008
The companies initially will seek to have the drug approved for treatment of people with familial hypercholesterolemia, a rare genetic disorder that causes exceptionally high levels of LDL cholesterol. Once the companies have shown the drug to be safe, tolerable and effective in that small market, they plan to expand trials to target increasingly larger markets, such as people who cannot control their cholesterol with other drugs. (San Diego Union-Tribune -- Business)
Genzyme Inks Deal With Isis On Cholesterol Drug Jan 8, 2008
It shows promise for all forms of high cholesterol, but the prospects are best in a rare disease called familial hypercholesterolemia (FH). The worst form, caused by having two bad copies of a particular cholesterol gene, afflicts only a few hundred people. (Forbes -- Business)
Genzyme Eyes Isis Drug Nov 10, 2007
Thursday, Isis announced trials that could get mipomersen approved in the rare genetic disease familial hypercholesterolemia (FH). The worst form, caused by having two bad copies of a particular cholesterol gene, afflicts only a few hundred people. (Forbes -- Business)
Carlsbad biotech goes on a tear Oct 14, 2007
It chose not to tackle the entire cholesterol market at once, preferring to try for the much smaller market for the genetic disease, called familial hypercholesterolemia ... Isis chose familial hypercholesterolemia as its first target because it is so severe and no effective treatment is on the market, McCamant said. (North County Times)
Screening Proposed for Childhood Cholesterol Levels Sep 15, 2007
THURSDAY, Sept. 13 (HealthDay News) -- A simple blood test early in life could identify children with familial hypercholesterolemia, a genetic condition that causes high cholesterol levels and greatly increases the risk of early death from heart disease, a British study finds ... "Our study shows that a test would identify 88 percent of cases of familial hypercholesterolemia, with only one in 1,000 misidentified," Wald said ... A study reported last month by cardiologists at the University of... (MEDLINEplus)
Statin Drugs May Delay Progressive Artery Damage In Children Aug 9, 2007
Starting statin therapy as young as age 8 safely and effectively delays the early artery damage caused by familial hypercholesterolemia (FH), according to a Dutch study reported in the August 7 print issue of Circulation: Journal of the American Heart Association. "Our data support early initiation of statin therapy in FH children, which might yield a larger benefit in the prevention of atherosclerosis later in life," said Barbara A. Hutten, Ph. (Science Daily)
Eight-year-olds 'could be given cholesterol drugs' Aug 7, 2007
They recommend the treatment only for those children with a genetic condition called familial hypercholesterolemia (FH), which causes very high levels of low density lipoprotein cholesterol, commonly called "bad" cholesterol, from birth onwards. Children with the condition show early signs of thickening of the artery walls and one in 20 has heart disease by the age of 30, with half suffering it by 50. (Independent)
Schering-Plough Announces ZETIA Approved in Japan for Treatment of High Cholesterol Apr 19, 2007
ZETIA is approved in Japan for use in patients with hypercholesterolemia, familial hypercholesterolemia or homozygous sitosterolemia. ZETIA can be used as a monotherapy and co- administered with a statin, for further reduction of LDL "bad" cholesterol in patients with hypercholesterolemia, familial hypercholesterolemia or homozygous sitosterolemia. (PR Newswire)
Monkey genes help us see what makes us human Apr 13, 2007
Jumping genes can cause inherited high cholesterol, also called familial hypercholesterolemia, breast cancer, hemophilia, and Tay-Sachs disease. "Who cares if a macaque has hypercholesterolemia, right? What we do care about is, hey, we have got these things in our own genomes that are constantly causing new mutations. Why are they doing it and how do we stop them?" Batzer asked. (Scientific American)
LSU professors monkey around with the Rhesus Macaque genome Apr 13, 2007
Mobile elements have also been shown to cause many diseases in humans such as breast cancer, familial hypercholesterolemia and Tay-Sachs and can also be involved in the creation of new genes and gene families. As a consequence, the impact of mobile elements on genomes in general, and on primate genomes in particular, is multifaceted. (EurekAlert!)
Study Presented at ACC Shows Treatment With ISIS 301012 Added to Statins for Only 5 Weeks Results in 75% of Patients Achieving LDL-C Levels Less Than 100 mg/dL and 50% of Patients Achieving LDL-C Levels of Less Than 70 mg/dL Mar 27, 2007
ALT Elevations Single(+) / Placebo 30 mg 100 mg 200 mg 300 mg 400 mg Consecutive(++) (n = 13) (n = 8) (n = 8) (n =16) (n = 8) (n = 8) Primary Endpoint (Day 1 to 59) 0 / 0 0 / 0 0 / 0 1 / 0 0 / 1 1 / 0 Primary Follow-up (Day 60 to 87) 0 / 0 0 / 0 0 / 0 0 / 1 0 / 0 0 / 0 Long term F/U (Day 88 to 199) 0 / 0 0 / 0 0 / 0 0 / 0 NA NA (+) Single = elevation that returned to below 3xULN by the next visit at least 7 days later (++) Consecutive = elevation that was observed in two measures at least 7 days... (PR Newswire)
Isis cholesterol drug shows promise in tests Mar 27, 2007
Another study was conducted in three patients with the genetic disorder familial hypercholesterolemia. In two cases, respective reductions in apoB-100, LDL-cholesterol and triglycerides of at least 50 percent were observed in a treatment regimen lasting at least 11 weeks. (North County Times)
Effect of Torcetrapib on Carotid Atherosclerosis in Familial Hypercholesterolemia Mar 27, 2007
Methods A total of 850 patients with heterozygous familial hypercholesterolemia underwent B-mode ultrasonography at baseline and at follow-up to measure changes in carotid intima media thickness ... Conclusions In patients with familial hypercholesterolemia, the use of torcetrapib with atorvastatin, as compared with atorvastatin alone, did not result in further reduction of progression of atherosclerosis, as assessed by a combined measure of carotid arterial-wall thickness, and was associated... (New England Journal of Medicine)
New Data Presented at ACC Show ISIS 301012 Monotherapy Reduces apoB to Undetectable Levels and Reduces LDL-C 70% Mar 26, 2007
ALT Elevations (++)Placebo 50 mg/wk 100 mg/wk Single(+) / Consecutive (++) (n = 10) (n = 8) (n = 8) Primary Endpoint Day 1 to 99 (78) 0 / 0 1 / 0 0 / 0 Primary Follow-up Day 100 to 143 0 / 0 0 / 0 0 / 0 Long term F/U Day 144 to 235 0 / 0 0 / 0 0 / 0 ALT Elevations 200 mg/wk 300 mg/wk 400 mg/wk Single(+) / Consecutive (++) (n = 8) (n = 8) (n = 8) Primary Endpoint Day 1 to 99 (78) 0 / 0 0 / 0 0 / 5* Primary Follow-up Day 100 to 143 0 / 0 1 / 0 NA Long term F/U Day 144 to 235 0 / 1 NA NA (+) Single... (PR Newswire)
Heart Association Backs Statin Use for At-Risk Kids Mar 24, 2007
"Guidelines drawn up by the National Cholesterol Education Program (NCEP) more than 10 years ago didn't really address the use of statins, a class of drugs that can lower cholesterol. Since that time, several drug trials in kids with familial hypercholesterolemia, a genetic predisposition to high cholesterol, have shown the use of statins had similar safety and effectiveness as in adults," Dr. Brian McCrindle, head of the statement writing group, said in a prepared statement ... The statement... (MEDLINEplus)
One Family's Gene Defect May Help Explain Heart Disease Mar 3, 2007
"If you look back on cholesterol, the work on familial hypercholesterolemia, which affects one person in a million, taught us about the relationship between cholesterol and heart disease," he said. "Studying these extreme outliers can be useful in the same way by identifying pathways that can be manipulated to treat or prevent metabolic syndrome.". (MEDLINEplus)
Experimental Drug Tackles Inherited High Cholesterol Jan 11, 2007
Dr. Daniel J. Rader, director of preventive cardiology at the University of Pennsylvania School of Medicine, and his colleagues gave the drug to six people with a condition called homozygous familial hypercholesterolemia (FH), in which a gene mutation makes cholesterol levels abnormally and dangerously high. Those with FH typically develop cholesterol problems early and may get heart disease before they reach the age of 20; they generally don't live past the age of 30. (Forbes)
Drug cuts lethal cholesterol levels Jan 11, 2007
The patients have a disease with a tongue-twister name, familial hypercholesterolemia (pronounced hyper-cholesterol-eemia), that afflicts about 300 people in the USA.. Patients with the disorder have "bad" cholesterol, or LDL, levels of 400 to 600 milligrams per deciliter of blood, more than quadruple the 130 recommended for healthy people. (USA Today)
Familial Hypercholesterolemia Jan 11, 2007
Please for full text and personal services. Marina Cuchel, M.D., Ph. (New England Journal of Medicine)
Penn researchers demonstrate ability of new therapy to treat severely elevated cholesterol levels Jan 11, 2007
In this study, patients with homozygous familial hypercholesterolemia (FH), a high-risk condition refractory to conventional therapy, had a remarkable 51% reduction in low-density lipoprotein (LDL) or "bad cholesterol" levels ... Rader concluded, "Although our study establishes proof of concept, a longer-term study in more patients will be required to determine the benefits and risks of this approach as a potential new therapy for homozygous familial hypercholesterolemia." Marina Cuchel, MD,... (EurekAlert!)
JCI table of contents: January 2, 2007 Jan 3, 2007
Similarly, humans with a mutation in the gene encoding ARH (who suffer from autosomal recessive hypercholesterolemia) are able to clear remnant lipoproteins from the blood, whereas individuals with mutations in their genes encoding the LDLR (who suffer from familial hypercholesterolemia) are not. In an accompanying commentary, Robert Mahley and Yadong Huang from the Gladstone Institute of Cardiovascular Disease discuss how these complex studies enhance our understanding of lipid clearance from... (EurekAlert!)
Pfizer's loss could be big gain for Carlsbad biotech Dec 5, 2006
Currently, 30-10-12 development is aimed at treating familial hypercholesterolemia, a common genetic disorder that makes sufferers more prone to heart attacks in early adulthood. If that proves successful, Isis plans to find a partner to help fund a treatment for more general cholesterol-related medical conditions. (North County Times)
Isis Pharmaceuticals Reports ISIS 301012 Significantly Reduces All Atherogenic Lipids When Dosed as a Single Agent and When Coadministered With Statins Nov 13, 2006
" ISIS 301012 Coadministered with Statins This randomized, double-blinded, placebo-controlled, dose-escalation study calls for five weeks of therapy at doses of 30, 100, 200, 300 and 400 mg/week. It was recently expanded to include longer-term treatment as well. Patients in the study have LDL-C levels between 100 and 220 mg/dL and have been on stable doses of / = 130 mg/dL) for three months with ISIS 301012 as a single agent. In April, results for the first three dose cohorts through 200 mg/week... (PR Newswire)
Ranbaxy Receives FDA Approval to Market Simvastatin 80mg Tablets with 180-Day Exclusivity Jun 24, 2006
Simvastatin tablets are indicated in the treatment of patients with coronary heart disease (CHD) or at high risk of CHD, reductions in risk of CHD mortality and cardiovascular events, patients with hypercholesterolemia requiring modifications of lipid profiles and adolescent patients with Heterozygous Familial Hypercholesterolemia (HeFH). A Simvastatin regimen can be started simultaneously with diet. (PR Newswire)
Pain in your heel? See a heart doctor Mar 15, 2006
Wider recognition of the link between Achilles tendon pain and so-called "heterozygous familial hypercholesterolemia" -- or HeFH -- could lead to earlier diagnosis of this disorder, the team reports in the Annals of Rheumatic Disease. Researcher Dr. Paul N. Durrington from the Manchester Royal Infirmary noted that HeFH is "the most common genetic disorder in Europe and the USA affecting 1 in 500 people.". (MSNBC -- Health)
Cholesterol Comeback Feb 7, 2006
301012 may have particular potential against a genetic disease called familial hypercholesterolemia (FH). In the worst cases, patients completely lack genes that instruct the liver to move cholesterol out of the blood. (Forbes -- Technology)
