Familial Hypercholesterolemia News and Research

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Genzyme, Isis receive 2014 Partners in Progress Corporate Award for KYNAMRO injection

Genzyme, a Sanofi company, and Isis Pharmaceuticals Inc., today announced that they have received the 2014 Partners in Progress Corporate Award from NORD in recognition of KYNAMRO (mipomersen sodium) injection.

9 May 2014

Controlling low-density lipoprotein-cholesterol levels: an interview with Jay Edelberg, M.D., Sanofi and Bill Sasiela, Ph.D., Regeneron

Lipids, primarily cholesterol and triglycerides, come from two sources: what is naturally manufactured in the liver and what comes from the intestines through the diet.

From Sanofi 7 May 2014

Synageva announces publication of LAL Deficiency review in Atherosclerosis

Synageva BioPharma Corp., a biopharmaceutical company developing therapeutic products for rare diseases, today announced the publication of an overview of lysosomal acid lipase deficiency (LAL Deficiency) in the online version and an upcoming print edition of Atherosclerosis, the official journal of the European Atherosclerosis Society.

21 Apr 2014

Clinical Research Forum announces winners of third annual Top 10 Clinical Research Achievement Awards

The Clinical Research Forum has announced the winners of its third annual Top 10 Clinical Research Achievement Awards. The winning projects represent the outstanding examples of research projects that benefit the health and well-being of the general public.

11 Apr 2014

Two Penn researchers selected as recipients of prestigious Clinical Research Achievement Award

Two researchers from the Perelman School of Medicine at the University of Pennsylvania and the Penn Cardiovascular Institute are among the 2014 recipients of the prestigious Clinical Research Achievement Award for their work in cardiovascular science.

11 Apr 2014

Amgen reports positive findings from AMG 145 Phase 3 studies in patients at risk for cardiovascular disease

Amgen (NASDAQ: AMGN) today announced new detailed data from three Phase 3 studies that showed treatment with its novel investigational cholesterol-lowering medication, evolocumab (AMG 145), resulted in a statistically significant reduction of 55-66 percent in low-density lipoprotein cholesterol (LDL-C) compared to placebo in patients with high cholesterol.

31 Mar 2014

Findings underscore importance of universal pediatric cholesterol screening

Roughly one out of three kids screened for high cholesterol between the ages of 9 and 11 has borderline or high cholesterol, potentially placing them at greater risk for future cardiovascular disease, according to research to be presented at the American College of Cardiology's 63rd Annual Scientific Session.

29 Mar 2014

Amgen announces results from Phase 3 TESLA Trial of evolocumab in patients with HoFH

Amgen (NASDAQ: AMGN) today announced that the Phase 3 TESLA (Trial Evaluating PCSK9 Antibody in Subjects with LDL Receptor Abnormalities) trial evaluating evolocumab met its primary endpoint of the percent reduction from baseline at week 12 in low-density lipoprotein cholesterol (LDL-C).

19 Mar 2014

Liposorber LA-15 System to treat pediatric patients with primary FSGS gets FDA approval

The U.S. Food and Drug Administration today approved Liposorber LA-15 System to treat pediatric patients with primary focal segmental glomerulosclerosis either before transplant, or after renal (kidney) transplantation in which there is recurrence of FSGS.

11 Oct 2013

Expert offers tips to help college students avoid serious health conditions down the road

To attract customers, restaurant chains have been rolling out budget deals, offering $5 pizzas, $3 meals — even $1 sandwiches. But while these new offerings are light on its customers' wallets, they hit them where it hurts in terms of calories, fat, and sodium content.

17 Sep 2013

Merck's LIPTRUZET tablets get FDA approval for treatment of elevated LDL cholesterol

Merck, known as MSD outside the United States and Canada, today announced that the U.S. Food and Drug Administration has approved LIPTRUZET (ezetimibe and atorvastatin) tablets for the treatment of elevated low-density lipoprotein cholesterol in patients with primary or mixed hyperlipidemia as adjunctive therapy to diet when diet alone is not enough.

4 May 2013

Investigational treatment for ALS passes early phase clinical trial for safety

An investigational treatment for an inherited form of Lou Gehrig's disease has passed an early phase clinical trial for safety, researchers at Washington University School of Medicine in St. Louis and Massachusetts General Hospital report.

24 Apr 2013

LDL apheresis can reduce cholesterol levels in patients with familial hypercholesterolemia

Some patients are genetically prone to such dangerously high levels of cholesterol that no amount of diet, exercise and medications can reduce their cholesterol to safe levels.

1 Mar 2013

Genetic testing strategy for familial high cholesterol questioned

Current testing strategies for the families of patients with familial hypercholesterolemia may not be an efficient use of health resources, suggest study results published in The Lancet.

25 Feb 2013

FDA approves Genzyme’s KYNAMRO to treat homozygous familial hypercholesterolemia

Genzyme, a Sanofi company, and Isis Pharmaceuticals Inc., today announced that the U.S. Food and Drug Administration (FDA) has approved its New Drug Application (NDA) for KYNAMROTM (mipomersen sodium) injection.

30 Jan 2013

Genzyme receives FDA approval for Kynamro to treat homozygous familial hypercholesterolemia

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).

30 Jan 2013

Juxtapid receives FDA approval for treatment of rare cholesterol disorder

On Dec. 21, the U.S. Food and Drug Administration approved Juxtapid (lomitapide) to reduce low-density lipoprotein (LDL) cholesterol, total cholesterol, apolipoprotein B, and non-high-density lipoprotein (non-HDL) cholesterol in patients with homozygous familial hypercholesterolemia (HoFH). Juxtapid is intended for use in combination with a low fat diet and other lipid-lowering treatments.

27 Dec 2012

Data from four Phase 2 trials of AMG 145 to be presented at AHA Scientific Sessions 2012

Amgen will present data from four Phase 2 trials of AMG 145, an investigational fully human monoclonal antibody directed against PCSK9, a protein that reduces the liver's ability to remove low density lipoprotein cholesterol, or "bad" cholesterol from the blood, at the upcoming American Heart Association Scientific Sessions 2012.

3 Nov 2012

Lomitapide shows promise in treating homozygous familial hypercholesterolemia

An international effort led by researchers at the Perelman School of Medicine at the University of Pennsylvania has resulted in positive phase 3 clinical trial results for a new medicine to treat patients suffering from a rare and deadly cholesterol disorder.

3 Nov 2012

University of Cambridge forms new spin-out company to supply hIPSC-derived cells

The potential therapeutic applications of stem cells - such as regenerating damaged tissues or organs - have generated a great deal of interest over the past decade. While these types of applications are exciting, it is a long journey from lab to clinic. The most immediate impact of stem cells on human health will most likely come from their use in the development of new drugs.

3 Nov 2012

Familial Hypercholesterolemia News and Research

Genzyme, Isis receive 2014 Partners in Progress Corporate Award for KYNAMRO injection

Controlling low-density lipoprotein-cholesterol levels: an interview with Jay Edelberg, M.D., Sanofi and Bill Sasiela, Ph.D., Regeneron

Synageva announces publication of LAL Deficiency review in Atherosclerosis

Clinical Research Forum announces winners of third annual Top 10 Clinical Research Achievement Awards

Two Penn researchers selected as recipients of prestigious Clinical Research Achievement Award

Amgen reports positive findings from AMG 145 Phase 3 studies in patients at risk for cardiovascular disease

Findings underscore importance of universal pediatric cholesterol screening

Amgen announces results from Phase 3 TESLA Trial of evolocumab in patients with HoFH

Liposorber LA-15 System to treat pediatric patients with primary FSGS gets FDA approval

Expert offers tips to help college students avoid serious health conditions down the road

Merck's LIPTRUZET tablets get FDA approval for treatment of elevated LDL cholesterol

Investigational treatment for ALS passes early phase clinical trial for safety

LDL apheresis can reduce cholesterol levels in patients with familial hypercholesterolemia

Genetic testing strategy for familial high cholesterol questioned

FDA approves Genzyme’s KYNAMRO to treat homozygous familial hypercholesterolemia

Genzyme receives FDA approval for Kynamro to treat homozygous familial hypercholesterolemia

Juxtapid receives FDA approval for treatment of rare cholesterol disorder

Data from four Phase 2 trials of AMG 145 to be presented at AHA Scientific Sessions 2012

Lomitapide shows promise in treating homozygous familial hypercholesterolemia

University of Cambridge forms new spin-out company to supply hIPSC-derived cells

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

From Puberty to Menopause: Clue’s CEO, Audrey Tsang on the Power of Femtech

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