Alert - rare disease
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Nils K Oeijord
May 7, 2013, 12:24:31 PM5/7/13
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| Viewpoint: Clear referral pathways needed for people with rare conditions GP online Dr Meech: 'It is vital that NHS England communicates with CCGs effectively on care needed by rare disease patients.' 1 April marked a dramatic shift in the way that healthcare will be delivered. For conditions such as Duchenne muscular dystrophy, which ... See all stories on this topic » | ||
| George West family raises awareness for rare disease through Relay for Life Corpus Christi Caller Times Their 21-month-old daughter was diagnosed in 2012 with severe combined immunodeficiency disease, often referred to as “bubble boy disease.” After a bone-marrow transplant last March, doctors were able to successfully treat it. “We're so thankful to ... See all stories on this topic » |
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Nils K Oeijord
May 7, 2013, 12:27:09 PM5/7/13
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| Rare Diseases Obscured by Shadows of 'Popular' Ills: Op-Ed Yahoo! News Without proper treatment, the more advanced my disease became, and the more disruptive my symptoms were. While the disease-specific details may differ, long diagnostic journeys are an unfortunate but inevitable reality for rare disease patients ... |
| Michael York battling rare disease RTE.ie York explained that he was originally diagnosed with bone cancer, before they discovered he actually had amyloidosis, a blood disease that breaks down tissues, leaving swollen growths and legions on the body. He told The Lady magazine: "It all began ... |
| CRF Raises $2.2 Million to Cure Cystinosis; Researchers Gain In Developing ... MarketWatch (press release) Cystinosis is a rare metabolic disease that causes early cell death in vital organs and afflicts about 2,000 people, mostly children, worldwide. "If this treatment works, it would be revolutionary and would change the course of corneal cystinosis ... |
| Dave Lemus Promoted to Chief Executive Officer of Sigma-Tau Pharmaceuticals ... Yahoo! New Zealand News Sigma-Tau Pharmaceuticals, Inc. is a U.S. based, wholly owned subsidiary of the Sigma-Tau Group, a member of the Global Rare Disease Franchise, and is dedicated to the global development and commercialization of medicines for patients with rare ... |
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Nils K Oeijord
May 7, 2013, 12:27:41 PM5/7/13
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| Fabry film spotlights a rare disease Medical Marketing and Media Genzyme is taking rare disease awareness to another level with a documentary film, dubbed “Facing Fabry Together,” that follows four families grappling with the genetic disorder. Genzyme manufactures Fabrazyme, an enzyme replacement therapy, for the ... |
| Wilcox Energy rallies for local girl with rare disease, CDKL5, for Team Avery Shoreline Times The members of Team Avery knew, as did Valvo, that a disease so rare that it afflicts only 400 girls worldwide — so rare that it has yet to be commonly identified by a proper name — has little chance of getting research funding, shifting the burden ... |
| FDA approves Procysbi for rare genetic condition FDA.gov The U.S. Food and Drug Administration today approved Procysbi (cysteamine bitartrate) for the management of nephropathic cystinosis in children and adults. Procysbi was granted orphan product designation because it is intended to treat a rare disease ... |
| Rare, lethal childhood disease tracked to protein - Medical Xpress Medical Xpress A team of international researchers led by Northwestern Medicine scientists has identified how a defective protein plays a central role in a rare, lethal childhood disease known as Giant Axonal Neuropathy, or GAN. The finding is reported in the May ... |
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| The Daily Struggle of a South Brunswick Child With a Rare Disease Patch.com When he was only 5-months-old, Ray was diagnosed with spinal muscular atrophy, an incurable, terminal disease that is the most frequent genetic cause of death for infants. The average life expectancy for a child with SMA is just two years, however Ray ... |
Nils K Oeijord
May 7, 2013, 12:26:13 PM5/7/13
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| Big Sur Marathon: Running eases pain for man with rare disorder Santa Cruz Sentinel Indeed, the 26.2-mile length of Sunday's Big Sur International Marathon probably will be only minimally daunting to Shane James, a 44-year-old Australian ultra-marathoner for whom running has become an indispensable weapon against an unusual disease ... |
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Nils K Oeijord
May 7, 2013, 12:28:19 PM5/7/13
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| FDA approves Raptor drug for rare disease North Bay Business Journal NOVATO — Raptor Pharmaceutical Corp. (Nasdaq: RPTP) today said the U.S. Food and Drug Administration approved its drug designed for the treatment of nephropathic cystinosis, a rare and possibly life-threatening metabolic disorder. This is the first ... |
| Raptor Drug Wins US Approval to Treat Rare Disease Bloomberg Raptor Pharmaceutical Corp. (RPTP) won U.S. approval for its drug to treat a rare metabolic disorder that attacks the kidneys. The drug called Procysbi, previously known as RP103, may generate sales of $77 million in 2015, according to the average of ... |
| Amherstburg child lives with rare genetic disorder Windsor Star (blog) Michelle, left, and Vince Laframboise with their children Rebecca, 6, and Connor, 3, at their Amherstburg home on Tuesday, April 30, 2013. Connor suffers from a rare disease and the family is holding a fundraiser to help buy a specialized van. |
| Girl fundraises while fighting rare disease Edmonton Examiner Young Stephie Gagnon's fight with a rare autoimmune disease has turned into a major fundraiser for the Stollery Children's Hospital. In 2009, at age six, Stephie was diagnosed with Juvenile Dermatomyositis (JDM), which caused painful damage in her ... |
| Raptor wins FDA approval of treatment for rare disease cystinosis San Francisco Business Times (blog) Raptor Pharmaceutical Inc. won Food and Drug approval Tuesday for its treatment for the rare, potentially fatal disease called cystinosis. The victory triggers a $25 million payment from HealthCare Royalty Partners and means Novato-based Raptor (NASDAQ ... |
| Genemarkers Proud Sponsor of the Rare Disease Challenge Wall Street Journal (press release) Genemarkers is proud to be a sponsor of the Rare Disease Science Challenge: Be HEARD (Helping Empower and Accelerate Research Discoveries) and work with Lori Silveira at National Jewish Health on her research project. Genemarkers will be ... |
| Sigma-Tau Pharmaceuticals Launches CYSTARAN(TM) for the Treatment of ... Yahoo! New Zealand News GAITHERSBURG, Md., May 1, 2013 (GLOBE NEWSWIRE) -- Sigma-Tau Pharmaceuticals, Inc. (Sigma-Tau), a part of the Sigma-Tau Group Rare Disease Franchise, announced today the availability of CYSTARAN(TM) (cysteamine ophthalmic solution) 0.44% ... |
| Ultragenyx Appoints Eric Yuen, MD as Chief Medical Officer and Senior Vice ... Yahoo! New Zealand News NOVATO, Calif., May 1, 2013 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on the development of treatments for rare and ultra-rare genetic disorders, today announced it has appointed Eric Yuen, MD as its Chief ... |
| FDA approves Raptor's Procysbi for rare genetic condition; and Plan B One-Step The Pharma Letter Procysbi was granted orphan product designation because it is intended to treat a rare disease or condition. Cystinosis affects an estimated 500 patients in the USA and about 3,000 patients worldwide. Fatal if not treated in early childhood, cystinosis ... |
| Celebrating May and Celiac awareness month Houston Chronicle (blog) Back then, celiac disease was considered a rare disease. Now, thanks to a prevalence study by Alessio Fasano, MD of the Center for Celiac Research and Treatment at Mass General Hospital, we know the incidence is much higher, 1 in 133 Americans. |
Nils K Oeijord
May 15, 2013, 1:36:39 PM5/15/13
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| Child fights for her life against rare disease Northern Virginia Daily From the end of July to September 6, Jordyn remained in the NICU where she was treated for a variety of issues including chronic lung disease, acidic blood and low glucose levels. It was also discovered that Jordyn had an extra chromosome - Trisomy 15q ... |
Nils K Oeijord
May 15, 2013, 1:34:39 PM5/15/13
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| Teen with rare disease comes home WCVB Boston Prosecutor: Kidnap suspect depraved. Text Size: ASmall Text; AMedium Text; ALarge Text. Theo Menswar goes home. A viral video prompted several doctors from across the country to come up with a treatment plan for a teen with a rare disease. |
| Horse visit wish granted for girl with rare disease Chicago Tribune Kira Mammoser, 9, meets Chicago Police horse "Mahoney" outside Advocate Children's hospital in Park Ridge on May 8, 2012. Kira, who has a rare disease, was granted her wish to see horses when two were brought to the hospital. (Antonio Perez, Tribune) ... |
| Shire signs rare disease pill pact with Nimbus PharmaTimes The partners are keeping their cards very close to their chest, saying that the collaboration is the result of a joint assessment of a series of rare disease targets "with significant unmet medical need [and] one target was ultimately chosen to be the ... |
| Downriver Girl With A Rare Disease Gets A Special Gift CBS Local DETROIT (WWJ) A Downriver girl with a rare disease got a special gift from a metro Detroit children's charity, Children With Hair Loss, on Wednesday. Lindsay Ratcliffe, 9, got a special custom-made wig. “Usually this is an unaffordable service to a ... |
| Girl battling rare disease gets a surprise visit Lake Forester Kira is battling a rare illness known as ROHHAD (Rapid-onset Obesity, with Hypothalamic dysfunction, Hypoventilation and Autonomic Dysregulation). She is one of only 75 people in the world known to be living with the disease, which has affected her ... |
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Nils K Oeijord
May 15, 2013, 1:36:03 PM5/15/13
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| National Organization for Rare Disorders to Celebrate 30 Years of Achievement ... Sacramento Bee WASHINGTON, May 10, 2013 -- /PRNewswire-USNewswire/ -- The 30th Anniversary Gala of the National Organization for Rare Disorders (NORD), to be held Tuesday, May 14, 2013, will honor rare disease heroes, celebrate 30 years of progress through the ... |
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| Children with rare disease CDG don't have mutation in every cell type HealthCanal.com Children born with rare, inherited conditions known as Congenital Disorders of Glycosylation, or CDG, have mutations in one of the many enzymes the body uses to decorate its proteins and cells with sugars. Properly diagnosing a child with CDG and ... |
| Lansing mom bravely battles rare disease Post-Tribune “One, because it becomes a pre-existing condition, and two, because stress progresses the disease. So, if I knew my daughters had it, I'd be under more stress. I'd constantly worry because they could have seizures and I'd be up all night worrying if ... |
| Nimbus inks co-development agreement with Shire to discover disease-altering ... pharmabiz.com Nimbus Discovery, LLC has entered into a co-development agreement with Shire plc focused on small molecule treatments for several rare genetic diseases known as lysosomal storage disorders (LSDs). The goal of the collaboration is to utilize the Nimbus ... |
| 4-Year-Old From South Africa Comes To LA To Beat Rare Disease CBS Local Months later, Reef contracted another rare disease: GVHD — “Grafts vs. Host Disease. The disease is a killer. You can die from it,” says Reef's mom, Lydia. He was going blind, deaf and his skin elasticity severely affected. “We were desperate. We had ... |
| French man rides bike across US to raise money for rare disease northwestohio.com The 62-year-old started his trip in New York City and is biking to raise money for research that will benefit his niece's daughter. She suffers from a rare genetic disorder called Wolfram Syndrome that causing diabetes, blindness and deafness. If you ... |
| FDA gives Novartis' Ilaris big boost with nod for rare juvenile diabetes FiercePharma Novartis ($NVS) has grabbed one of two new indications it was shooting for with its rare disease drug Ilaris, one step toward turning the drug into a blockbuster. The FDA approved Ilaris for treatment of SJIA, making it the only treatment for that ... |
| Genetic mutation during development causes port-wine stains and Sturge ... HealthCanal.com The Brain Vascular Malformation Consortium is part of the National Institutes of Health Rare Disease Clinical Research Network, supported through the NIH Office of Rare Diseases Research at the National Center for Advancing Translational Science and ... |
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