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News at a Glance

See articles regarding Gaucher disease and other rare diseases, health and nutrition, science and technology, Gaucher studies and more.

 

Crossing the Blood Brain Barrier

Jul 22, 2015 - Category: Health and Nutrition News

Crossing the Blood Brain BarrierTreating an aggressive brain tumor may have come one step closer to reality recently as a company developing a novel therapy got a grant. According to Irwin Hollander, Ph.D., vice president for research and development at Lauren Sciences, "V-Smart™ for GBM, formulated with a potent anti-tumor agent, has promise as an effective new treatment for GBM patients. [...]

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Woman suffers rare disease after taking over-the-counter drug

Jul 21, 2015 - Category: Rare And Orphan Disease News

The Food and Drug Administration has recently asked for new warnings on painkillers like ibuprofen, saying these over-the-counter drugs do raise the risk of heart attack or stroke. But a different side-effect recently landed a woman in the burn unit at Vanderbilt. [...]

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Young Boy Inspires Documentary Series

Jul 21, 2015 - Category: Rare And Orphan Disease News

Currently, they are working on a new series for TLC about people with rare genetic conditions. The documentary series will feature inspirational adults and children as well as, in some cases, their parents, carers, close family and friends. [...]

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How can these scientists get their promising new drug to market?

Jul 21, 2015 - Category: Rare And Orphan Disease News

How can these scientists get their promising new drug to market?Katharine Sepp, chief executive officer, and Joost Schulte, chief scientific officer, co-founders of Oxalys Pharma, knew that they had discovered a promising new drug candidate for treating neurodegenerative disorders such as Parkinson’s and Huntington diseases but the challenge was to figure out a way to successfully commercialize their discovery. [...]

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Nutrition: Banning trans fats is a healthy change

Jul 21, 2015 - Category: Health and Nutrition News

Nutrition: Banning trans fats is a healthy changeThe U.S. Food and Drug Administration recently announced that partially hydrogenated oils or trans fats are no longer considered safe and will be banned from all foods in three years. Trans fats lower good HDL cholesterol, raise bad LDL cholesterol and triglycerides, increase insulin resistance and increase inflammation in our bodies. [...]

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Parents seeks treatment for 4-year-old son's rare illness which causes stomach to swell

Jul 21, 2015 - Category: Gaucher Disease News

Parents seeks treatment for 4-year-old son's rare illness which causes stomach to swellThe parents of a four-year-old boy who was diagnosed with a rare illness causing his stomach to swell to the size of a basketball are now seeking medical treatment to cure him. Young Niu Shaolong is from a small village in Zuoquan county, Shanxi. His parents, both farmers, have been doing everything they can to give the boy the help he needs. [...]

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(EACT), S 139/HR 209 letter from CFF to rare disease partners

Jul 20, 2015 - Category: Legislative News

Several months ago you joined with the Cystic Fibrosis Foundation and others in signing a letter in support of the Ensuring Access to Clinical Trials Act (EACT), S 139/HR 209, to permanently remove a barrier to clinical trial participation for those with rare diseases. [...]

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High-risk world of rare genetic diseases: Research efforts improving

Jul 19, 2015 - Category: Rare And Orphan Disease News

High-risk world of rare genetic diseases: Research efforts improvingThese are not isolated stories. In the United States and Europe, a familiar narrative is emerging—a wealthy family takes a child in for a checkup, concerned about her development. Upon hearing a diagnosis of a rare disease, the family flexes its financial resources to try to direct research to cure the disorder. [...]

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Cerdelga Patient Webcast

Jul 17, 2015 - Category: Gaucher Disease News

Patient Interview and webcast with Dr. Pram Mistry. In this webcast patients share their treatment experiences. Dr. Mistry answers some of the most commonly asked questions about Ceredelga. [...]

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How Climate Change Could Threaten The Nutrition Of Millions

Jul 16, 2015 - Category: Health and Nutrition News

How Climate Change Could Threaten The Nutrition Of MillionsThe world already has a hard time properly allocating crucial nutrients to its 7.125 billion residents — and a new study published in The Lancet Wednesday suggests that global warming is only going to increase that challenge. According to the World Health Organization, zinc deficiency currently impacts a little more than thirty percent of humans across the globe. [...]

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The Cystic Fibrosis Foundation Applauds Passage of the Ensuring Access to Clinical Trials Act of 2015 in the U.S. Senate

Jul 16, 2015 - Category: Rare And Orphan Disease News

The Cystic Fibrosis Foundation applauds passage of a bill today in the United States Senate to remove barriers to participation in clinical trials for much-needed treatments of rare diseases or conditions, including cystic fibrosis. [...]

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Celebrate Your Victory – the 21st Century Cures Act Passes the House!

Jul 11, 2015 - Category: Rare And Orphan Disease News

Celebrate Your Victory – the 21st Century Cures Act Passes the House!You Helped Make it Happen! Rare disease patients, advocates and families across the country, including Global Genes and our network, united to make this happen! Your voices were heard by Congress and the House passage of this landmark legislation is an example of what the rare community can accomplish when we work together. [...]

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Desperately ill Alrene yet to receive medication

Jul 8, 2015 - Category: Gaucher Disease News

Desperately ill Alrene yet to receive medicationTHE life of a critically ill 5-year-old girl from Booysens Park hangs in the balance as her parents await the outcome of the government health department "procurement procedures" for the medication she needs. Alrene Scheepers has been waiting for 19 months for her much needed medication to finally be purchased by Dora Nginza Hospital. [...]

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Forcing food companies to cut salt levels could save 4,500 lives in the next decade, scientists say

Jul 6, 2015 - Category: Health and Nutrition News

The health foods that are not that healthyTougher compulsory limits on salt in food would save three times as many lives from heart disease, new research found. It would slash cardiac deaths by about 4,500 in the next decade, compared to 1,500 under the current voluntary pledges by manufacturers to cut salt in processed foods. [...]

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The health foods that are not that healthy

Jul 3, 2015 - Category: Health and Nutrition News

The health foods that are not that healthyIn life, often if it seems too good to be true, it usually is and unfortunately this can be said for some of our favourite 'health' foods. Here are some of the most common 'not so healthy' offenders for you so you can be sure that your healthy choices really are all that you need them to be to keep your body in tip top shape. [...]

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The Surprising Role of Nutrition in Mental Health

Jul 1, 2015 - Category: Health and Nutrition News

If you’ve been reading my blog for a month or more, you know that I have found nutrition to be a powerful force in my recovery from depression. [...]

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Little girl gets go-ahead for expensive treatment

Jul 1, 2015 - Category: Gaucher Disease News

Little girl gets go-ahead for expensive treatmentFive-year-old Alrene suffers from type 1 Gaucher’s disease. It’s a chronic illness that according to WebMD.com is caused by an enzyme not working right to break down a certain type of fat. The fat then builds up, especially in the liver and spleen. The only available treatment for Alrene costs thousands of rands. [...]

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Three simple ways to improve your health

Jul 1, 2015 - Category: Health and Nutrition News

Three simple ways to improve your healthWhen people around North Texas think about losing weight and gaining health, their minds inevitably think of diets, deprivation and excessive exercise. For too many, healthy living and eating is perceived as difficult disengaging, bland and boring. "When we engage in something, we attract it," says Cindy Carbone of Southlake-based Commit to H.E.A.L. [...]

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Half of All Heart Disease Deaths Are Preventable

Jun 30, 2015 - Category: Health and Nutrition News

Half of All Heart Disease Deaths Are PreventableHalf of All Heart Disease Deaths Are Preventable Experts hope that raising awareness will help lower the death rate. In the U.S., preventable risk factors still account for 50 percent of deaths from cardiovascular disease among adults age 45 to 79, according to a new analysis. But even if every state brought levels of those risk factors. [...]

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Shire's "Spotlight on Gaucher" Film Contest

Jun 30, 2015 - Category: Gaucher Disease News

Shire's "Spotlight on Gaucher" Film ContestShare your own personal story of living with Gaucher disease. We invite you to make a video about life with or your experiences of Gaucher disease. Sharing your personal experience may empower others to overcome the challenges of living with the condition, while also raising awareness of the signs and symptoms of Gaucher disease. [...]

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Response to King v. Burwell Decision

Jun 25, 2015 - Category: Legislative News

Response to King v. Burwell DecisionToday’s Supreme Court decision is a victory for millions of Americans who have gained health insurance under the Affordable Care Act and who are at reduced risk of losing everything to the physical and financial costs of the injuries and illnesses we all experience. In this way, today’s decision promotes effective public policy against homelessness. [...]

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The Government Tells You How to Eat Healthy, But Should It Tell You How to Eat Green?

Jun 24, 2015 - Category: Health and Nutrition News

The Government Tells You How to Eat Healthy, But Should It Tell You How to Eat Green?Eating less meat is better for the environment.That's the advice doled out by an independent panel of experts earlier this year in a set of recommendations for the next round of federal dietary guidelines. But if House Republicans get their way, that message may never be delivered. [...]

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Advances in Understanding and Treatment of Gaucher Disease

Jun 24, 2015 - Category: Gaucher Disease News

Dr. Mistry spoke on new advances in understanding the role of the immune system in Gaucher disease and treatment of Gaucher disease. He covered the FDA approved treatments of Gaucher disease in his talk and discussed unmet needs. [...]

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West discovers health benefits of Indian spice turmeric

Jun 22, 2015 - Category: Health and Nutrition News

West discovers health benefits of Indian spice turmericMultiple studies show that turmeric can help prevent or treat a wide spectrum of cancers, inflammatory conditions, autoimmune problems, neurological ailments including Alzheimer's disease, cardiovascular disease, diabetes and diabetes neuropathy, among other metabolic diseases. [...]

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Switching from imiglucerase to miglustat for the treatment of French patients with Gaucher disease type 1: a case series

Jun 22, 2015 - Category: Gaucher Disease News

Treatment with enzyme replacement therapy has been available for the past two decades but, although effective, enzyme replacement therapy can be delivered only by intravenous infusion every other week. [...]

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Guest opinion: Thousands of local kids fall into summer nutrition gap

Jun 20, 2015 - Category: Health and Nutrition News

While adults may reminisce about carefree childhood summers, this time of year means fewer nutrition resources for many kids across California. [...]

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Biologists Invoke the Past in Modern Bacteria By swapping ancient genes into modern E. coli, scientists hope to tease out the rules of evolution.

Jun 18, 2015 - Category: Health and Nutrition News

Biologists Invoke the Past in Modern Bacteria By swapping ancient genes into modern E. coliOne of the greatest challenges in evolutionary biology is trying to piece together the history of life based on fragmentary evidence and limited knowledge of the forces at play — the rich tapestry of climate, geology and living things that forms the backdrop for evolution. How, for example, did the light-sensitive molecules that enable color vision come into being? [...]

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New White Paper from BIO, NORD, and EY Finds Orphan Drug Tax Credit is Crucial to Rare Disease R&D

Jun 17, 2015 - Category: Rare And Orphan Disease News

Today, the Biotechnology Industry Organization (BIO), the National Organization for Rare Disorders (NORD), and Ernst & Young released "Impact of the Orphan Drug Tax Credit on Treatments for Rare Diseases". [...]

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Canada's Rare Disease Strategy Needs to Become a Reality

Jun 17, 2015 - Category: Rare And Orphan Disease News

Canada's Rare Disease Strategy Needs to Become a Reality Kate is an energetic 24-year-old Ottawa woman who describes herself as an "all too rare, rare disease success story." She and her older sister Meghan have been diagnosed with type 1 Gaucher disease -- a debilitating and painful illness which causes a damaging buildup of fatty substances in vital organs. [...]

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TS govt told to bear expenses of ailing girl

Jun 17, 2015 - Category: Gaucher Disease News

TS govt told to bear expenses of ailing girlTelangana, India Justice PV Sanjay Kumar of the High Court at Hyderabad​ in India on Tuesday directed the state government to undertake for the present the expenses and ensure treatment to three-year- old Sammeeksha who is suffering from a rare medical disorder called the Gaucher disease. [...]

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FDA bans artificial trans fats

Jun 16, 2015 - Category: Health and Nutrition News

FDA bans artificial trans fatsThe U.S. Food and Drug Administration took a key step toward a complete ban on artificial trans fats Tuesday, saying that partially hydrogenated oils are not "generally recognized as safe," and giving food companies three years to remove artificial trans fats from their products. [...]

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Nutrition and Mental Health: Serving Up Happier Days

Jun 11, 2015 - Category: Health and Nutrition News

Nourish your sense of accomplishment and let this fuel each step towards your personal recovery. Do what you can with what you have while you’re getting where you want to be. [...]

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Nutrition is Vital for Mental Health

Jun 11, 2015 - Category: Health and Nutrition News

Poor nutrition is often an underlying factor and aggravator behind many types of mental illnesses, including depression, according to a new international study involving the faculty of medicine and dentistry of the University of Valencia in Spain. [...]

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Missing link found between brain, immune system; major disease implications

Jun 1, 2015 - Category: Health and Nutrition News

In a stunning discovery that overturns decades of textbook teaching, researchers at the University of Virginia School of Medicine have determined that the brain is directly connected to the immune system by vessels previously thought not to exist. [...]

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Rare Families Needed to be matched with Medical Students

Jun 1, 2015 - Category: Rare And Orphan Disease News

Earlier this month, we launched our 2nd annual Cox Prize for Rare Compassion contest which encourages 1st and 2nd year medical students to get to know a rare family and write an essay based on their experiences. [...]

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