• to account for 19 percent of worldwide prescription sales at a value of $176 billion by 2020, and with typically low commercialization costs, rare disease therapies are becoming increasingly attractive and are expected to further bring transformational patient benefits. (mckinsey.com)
  • Rare diseases: how can we improve diagnosis and treatment? (weforum.org)
  • Patients affected by rare diseases often find there are no treatments for their condition. (mckinsey.com)
  • Rare diseases by definition affect a small number of patients, and they historically have not attracted significant pharmaceutical investment. (mckinsey.com)
  • Recently, though, large pharmaceutical companies have begun to pay more attention to rare diseases, drawn by government incentives and the greater likelihood that treatments for what are often life-threatening or severely debilitating diseases will be successful for both pharmacos and for patients. (mckinsey.com)
  • And they help patients and their caregivers navigate a healthcare system not usually geared to supporting those with rare diseases. (mckinsey.com)
  • BOSTON and ALISO VIEJO, Calif. , Aug. 18, 2016 /PRNewswire-USNewswire/ -- Global Genes , a leading global advocacy organization for patients and families fighting rare and genetic diseases, and PlatformQ Health are proud to announce the strategic partnership to develop a first of it's kind educational platform, RareUniversity. (prnewswire.com)
  • Together rare diseases define a group of over 7,300 ailments from Alzheimers to Zygomycosis. (prnewswire.com)
  • Global Genes is a leading global advocacy organization for patients and families fighting rare and genetic diseases. (prnewswire.com)
  • More than 300 million people worldwide live with rare diseases, for which there are no preventative measures, no cures, and no effective treatments. (weforum.org)
  • All together, rare diseases have a huge impact on global health. (weforum.org)
  • A new initiative to boost research and development into rare and paediatric diseases will launch today at the European Health Summit. (europabio.org)
  • the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE), the European Clinical Research Infrastructure Network (ECRIN), EuropaBio, EURORDIS-Rare Diseases Europe, the European Federation of Pharmaceutical Industries and Associations (EFPIA) and the European Infrastructure for Translational Medicine (EATRIS). (europabio.org)
  • The coalition says it is vital to speed up research into more than 7000 identified rare diseases, of which a staggering 95 per cent have no approved treatment or cure. (europabio.org)
  • The Rare Disease Moonshot is also a response to the European Commission's call to address unmet medical needs - and the belief that the science and translational capability gap in rare diseases cannot be addressed by one party or even one sector alone, nor can it be optimally served by a "patchwork" of unrelated initiatives. (europabio.org)
  • While the journey to its shared goal of delivering new therapies for rare diseases where no treatment option exists is long, the coalition is confident this initiative can take us one step closer. (europabio.org)
  • In addition to Dr. Clemons, the centre will be led by a team of senior industry executive experts in rare diseases including Thomas Ogorka (Managing Director) and Nazira Maruf (Vice President of Project Management). (ddw-online.com)
  • On that day, millions of patients and their families around the world share their stories in order to raise awareness about rare diseases and their impact. (cdc.gov)
  • There are thousands of diseases that are individually rare but collectively common. (cdc.gov)
  • In the United States, a rare disease is defined as a condition that affects fewer than 200,000 people, and rare diseases as a whole affect about 25 million people in the United States, and about 400 million worldwide. (cdc.gov)
  • Examples of rare diseases include Huntington disease, fragile X syndrome, Guillain-Barré syndrome, Crohn disease, and Duchenne muscular dystrophy. (cdc.gov)
  • In addition, the economic impact of rare diseases is substantial not only for affected patients and their families, but for society as a whole. (cdc.gov)
  • What does genomics have to do with rare diseases? (cdc.gov)
  • Most rare diseases have a genetic and/or congenital cause. (cdc.gov)
  • To date, more than 7,000 rare diseases have been described, although the precise genetic causes for many of them remain unknown. (cdc.gov)
  • This technology has provided success in identifying new causal mutations for rare suspected genetic diseases of previously unknown cause, with diagnostic rates of 25%-50% in recent studies . (cdc.gov)
  • Molecular genomic diagnosis of rare diseases can lead to changes in medical care including use of existing medications or development of new ones to help people with specific mutations, as well as discontinuing ineffective therapies. (cdc.gov)
  • Molecular insights have not led to effective therapies for most rare diseases so far, but they do promise deeper understanding into the biology of these conditions that could lead to better management and improved outcomes in the future. (cdc.gov)
  • For an increasing number of rare diseases, treatments are becoming readily available, which can ameliorate or reduce the burden of illness and delay death. (cdc.gov)
  • Public health is no stranger to the world of rare and genetic diseases. (cdc.gov)
  • It was regarded as a problem of the developed world where it is only second to cardiovascular diseases as a cause of death. (who.int)
  • Rare Disease PHGKB is an online, continuously updated, searchable database of published scientific literature, CDC and NIH resources, and other information that address the public health impact and translation of genomic and other precision health discoveries into improved health outcomes related to rare diseases. (cdc.gov)
  • We report a sentinel case of acute eosinophilic pneumonia in a firefighter exposed to high concentrations of World Trade Center dust during the rescue effort from September 11 to 24. (cdc.gov)
  • Firefighters participating in rescue/recovery work at the World Trade Center site lost an average of ~10% of FEV1 in the first post-9/11 year, with little apparent recovery (on average) over the next 6 years. (cdc.gov)
  • Dr. Christine Dingivan, Chief Executive Officer of Emmes, added: "We want to make it easier for both rare disease focused biopharma companies and public sector research institutions to accelerate development of new treatment options for rare disease patients by leveraging a highly specialised global CRO committed to rare disease clinical research. (ddw-online.com)
  • Those paying attention to the news may note that the governments of the world recently toyed with the scenario of a global monkeypox pandemic. (rt.com)
  • Gun crime is very rare in South Korea and the incident in the southern city of Sejong, 120 kilometres (75 miles) south of Seoul, received blanket coverage on TV news stations. (hurriyetdailynews.com)
  • The ' Rare Disease Moonshot ' is a commitment and collaboration between seven organisations to break down the barriers to finding new treatments and cures for the world's rarest and severe conditions which currently have no therapeutic options, and which often affect the youngest patients. (europabio.org)
  • Thanks to the "new lake" effect that saw rainfall wipe out several years of severe drought, Ivie-always a big-bass producer since the 19,149-acre water body was impounded back in 1990-is greatly re-energized as water levels have turned acres of brush, scrubby timber and terrestrial vegetation into a cover-filled Disney World for bucketmouth bass. (gameandfishmag.com)
  • Severe side effects are extremely rare. (bvsalud.org)
  • World Orphan Drug Conference 2018, April 25-27 (Part 1) Getting to the Conference! (cysticfibrosis.com)
  • Evidence discovered in 2018 that both daughters carried a rare CALM2 genetic variant was one of the reasons that an inquiry was called a near later - but which found no grounds for reasonable doubt. (sky.com)
  • But when it was discovered in 2018 that Sarah and Lara had carried the rare CALM2 genetic variant, the original inquiry into the convictions was launched. (sky.com)
  • In the meantime, Frankens-a May 2018 business and marketing graduate from Stephen F. Austin State University in Nacogdoches-will be thankful and content for the way he's already found bass-fishing fame, thanks to a world-record fishing trip visit a few weeks ago to Texas' red-hot O.H. Ivie Lake . (gameandfishmag.com)
  • (http://www.un.org/en/sections/observances/international-days/, accessed 27 September 2018). (who.int)
  • GWD is rare, with only three remaining human cases left in the world, as of March 2018. (cdc.gov)
  • The global price for rare earths skyrocketed, and there was a fair bit of alarm in the US about how China's chokehold on rare earths threatened the economy and even national security. (vox.com)
  • Not long after China restricted exports, other countries quickly began producing their own rare earths - or finding ways to reduce their reliance on the metals. (vox.com)
  • Next, many Japanese companies rushed to reduce their heavy reliance on rare earths. (vox.com)
  • Finally, Gholz notes, it turned out that China's export curbs weren't fully effective, particularly for heavy rare earths such as dysprosium and europium. (vox.com)
  • In the end, Gholz argues, China didn't get that much benefit from restricting rare earths - save for the release of a fishing captain who had been detained by Japan. (vox.com)
  • Japan has now adjusted and is less vulnerable to trade pressure over rare earths than once believed. (vox.com)
  • A few years ago, military planners had worried that crucial weapons systems might be at risk if China disrupted the rare earths supply. (vox.com)
  • China's grip over the world's supply of rare-earth metals has weakened considerably - and the country can't hold the global economy hostage, as was once widely feared. (vox.com)
  • The United States, too, managed to wiggle out of China's rare-earth grip in short order. (vox.com)
  • The newly dedicated centre will be launched at the World Orphan Drug Congress in Barcelona, Spain on November 15. (ddw-online.com)
  • The effect has been that, in 2015, 45 novel rare disease therapies were approved by the FDA's Center for Drug Evaluation and Research, significantly more than the average of 28 approved during each of the previous nine years. (mckinsey.com)
  • Green is the rarest eye color in the world, with only 2% of the world's population (and fewer than one out of ten Americans) sporting green peepers, according to the American Academy of Ophthalmology (AAO). (rd.com)
  • A new study suggests that complex alien life -- the sort of life we could one day meet and potentially communicate with -- might be present on far fewer worlds than previously thought, due to the toxic presence of so much CO 2 on certain planets that are otherwise within the Habitable Zone (HZ) of their respective stars. (extremetech.com)
  • What is World AIDS Day and why does it matter? (weforum.org)
  • How is the World Economic Forum bringing data-driven healthcare to life? (weforum.org)
  • World Economic Forum articles may be republished in accordance with the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International Public License, and in accordance with our Terms of Use. (weforum.org)
  • The views expressed in this article are those of the author alone and not the World Economic Forum. (weforum.org)
  • Traci Clemons, Chief Research Officer of Emmes, said: "Collectively, we have an incredibly strong team, bringing together deep global rare disease clinical operations capabilities with extensive scientific research experience across multiple rare disease indications. (ddw-online.com)
  • Still, since 1950, when the rebbe sent out his first emissary to Morocco to build up Jewish education in North Africa, some 4,000 have dispersed around the world, from Beijing to Ukraine to Laos to Boise, Idaho, and virtually every spot in between, where Jews might live or pass through. (jta.org)
  • Until recently, cancer was thought to be rare in Africa. (who.int)
  • however, the disease is still common today in other parts of the world, including the Indian subcontinent and sub-Saharan Africa. (cdc.gov)
  • Mycobacterium africanum is endemic to West Africa and is rare outside this region. (who.int)
  • We report a rare case of pulmonary tuberculosis (TB) secondary to M. africanum in a man in Brunei Darussalam who had lived and worked in Guinea, West Africa for 6 years more than 20 years ago. (who.int)
  • While all drug launches are complex, launches of rare disease treatments are particularly so. (mckinsey.com)
  • Our experience suggests that companies that launch rare disease treatments must excel in four areas. (mckinsey.com)
  • Our experience suggests that companies that launch rare disease treatments successfully excel in four areas. (mckinsey.com)
  • Experts say that at the current pace of R&D it could take more than 100 years to find treatments for all current rare conditions. (europabio.org)
  • The World Health Assembly has established mandates regarding the observance of international anniversaries through resolutions. (who.int)
  • These are endorsed by the World Health Assembly resolution (WHA58.22) on cancer prevention and control. (who.int)
  • The expanded Orphan Reach is dedicated to the advancement of rare disease research by providing specialised clinical trial design and execution capabilities. (ddw-online.com)
  • It is expected that the Orphan Reach clinical study portfolio will double over the next 12 months, with Emmes' joint services and technologies bringing new capabilities to the expanding rare disease drug development market. (ddw-online.com)
  • Case report: The current manuscript highlights clinical and radiographic features of a rare case of PIOC in a 32 year old Indian female patient. (bvsalud.org)
  • They used to be abundant across the Atlantic and Mediterranean seas, but after years of habitat disturbance, pollution and incidental catch in fisheries, they are among the rarest shark species in the world. (dailypost.co.uk)
  • The world's rarest duck, the Madagascar pochard, has taken a small step away from the threat of extinction, thanks to a conservation project that has received support from the Mohamed bin Zayed Species Conservation Fund. (emirates247.com)
  • Determined to conserve this unique bear species and its habitat, India-based wildlife conservation charity Wildlife SOS in collaboration with the IUCN-SSC Sloth Bear Expert Team has declared 12th October as World Sloth Bear Day. (indiatimes.com)
  • On that day, March 1, Frankens caught an uncommon record, a 7-pound, 9-ounce smallmouth/largemouth bass hybrid that has been recognized by the International Game Fish Association as an all-tackle world record for the species . (gameandfishmag.com)
  • The last place we visit is a cheese bank, a vault where the forms age for six or seven years, or even more, transforming Bitto into one of the world's rarest cheeses. (slowfood.com)
  • Unfortunately, the flower is one of the rarest in the world, as it's been extinct in the wild for over 100 years now. (apartmenttherapy.com)
  • In "universe years," that's merely childhood, which is certainly incredible. (diyphotography.net)
  • The current age of our universe is 13.8 billion years, and the latest supernova image was shot when it was less than a fifth of its current age. (diyphotography.net)
  • Only produced and released in the finest years of production, 1800® Colección is one of the rarest tequilas in the world. (1800tequila.com)
  • The rare earth shortage quickly resolved itself within a few years. (vox.com)
  • Michael Egan is an expert in fine and rare wines with more than thirty-five years of experience. (citizen.co.za)
  • Thanks to many years spent inspecting old and rare wines in European cellars for auction, where in many instances the wines had lain undisturbed since original purchase shortly after bottling, he is in the fortunate position to know what a genuine bottle should look like, hence his specialty in detecting counterfeit bottles. (citizen.co.za)
  • Twenty years on, a professor has named the "rare" condition that her daughters had. (sky.com)
  • New plasma coating technology could see the phase-out of rare earth metal indium that is used in smartphone glass and dimmable windows, which is predicted to run out in 10 years. (edu.au)
  • It's been more than 9 years, and we've got patients all over the world who are now on it. (medscape.com)
  • The list of 31 African countries can be found on the World Health Organization (WHO) Onchocerciasis Fact Sheet external icon external icon website. (cdc.gov)
  • Four countries have been verified by the World Health Organization as free from onchocerciasis: Colombia, Ecuador, Mexico, and Guatemala. (cdc.gov)
  • However, the worldwide burden of onchocerciasis has been considerably reduced as the result of very successful disease control programs led by the World Health Organization (WHO). (cdc.gov)
  • The Pan American Health Organization / World Health Organization (PAHO / WHO) estimates that the most important measure to prevent yellow fever is immunization. (bvsalud.org)
  • Entitled, "Brown Eye Blue", this picture was captured in a remote village, in rural Chad, along the Chari River, while conducting a study with the Carter Center, and the World Health Organization (WHO). (cdc.gov)
  • In 1991, after a systematic review of its histology and degree of differentiation, the World Health Organization (WHO) classification recommended that the term "mucoepidermoid tumor" be changed to "mucoepidermoid carcinoma" 11 . (bvsalud.org)
  • This is also the first time astronomers were able to measure the size of a dying star in the early universe," writes NASA. (diyphotography.net)
  • NEW JERSEY - NASA astronaut Scott Kelly captured a rare and spectacular display of 'thundersnow' from space as Snowzilla's blast pummeled much of the US East Coast this weekend with two feet or more of paralyzing snow from the nations' capital to New York City and beyond. (universetoday.com)
  • For more than half a century, newborn screening for selected metabolic and other rare genetic conditions has been a major public health program in the United States and many countries around the world. (cdc.gov)
  • Global Genes and PlatformQ Health are proud to partner together to overcome this fundamental challenge, launching an interactive online rare disease medical education platform - RareUniversity. (prnewswire.com)
  • Together with Global Genes and the organizations they serve, RareUniversity will provide a trusted educational platform to measurably improve the state of rare disease education and advocacy. (prnewswire.com)
  • Global Genes works to eliminate rare disease challenges by building awareness, developing patient-focused educational tools, and funding patient care programs and early investigative research. (prnewswire.com)
  • It is incredibly rare, this particular variant has not been found before in the world, but it occurs in three genes that together cause a condition known as calmodulinopathy," she said. (sky.com)
  • One of the world's rarest sharks has been found washed up on an Abersoch beach. (dailypost.co.uk)
  • One of these rare sharks was found on Traeth Trwyn Y Fach, near the Warren resort, last week. (dailypost.co.uk)
  • British scientists found an "incredibly rare" genetic mutation could have been responsible for the deaths of two children after their mother, who was originally jailed for killing them, has now been pardoned. (sky.com)
  • World Trade Center dust consists of large particle-size silicates, but fly ash and asbestos fibers may be found in bronchoalveolar lavage cells. (cdc.gov)
  • Acute eosinophilic pneumonia is a rare consequence of acute high dust exposure. (cdc.gov)
  • RareUniversity.com will launch in conjunction with the Fifth Annual RARE Patient Advocacy Summit, September 22-23, 2016. (prnewswire.com)
  • Brucellosis is a common zoonosis endemic in many parts of the world [1]. (who.int)
  • Accurate and clinically valid education, including reporting on breakthroughs in research and development, is one of the most valuable tools anyone facing a rare disease can have," says Robert Rosenbloom , CEO of PlatformQ Health. (prnewswire.com)
  • By understanding and supporting rare disease, we will prevail and make this world, and its' health, better for all of those that come after us. (rarediseaseday.org)
  • Emmes has launched a rare disease centre, Orphan Reach, to combine its health research across the public sector and biopharmaceutical industry, with Orphan Reach, a UK-based CRO, acquired by Emmes in May 2021. (ddw-online.com)
  • At two meetings held in October and November 2017, respectively, to consider, among other things, the provisional agenda of the Executive Board's 142nd session, the Officers of the Board discussed the subject of World Health Days. (who.int)
  • Action-oriented and comprehensive knowledge, relating health to-lifestyle, were rare. (lu.se)
  • These are the conditions in which the world's rarest books were born. (patreon.com)
  • Because the condition is rare, the launch team will have few, if any, analogs from which to draw lessons. (mckinsey.com)
  • The condition is rare in the United States. (medlineplus.gov)
  • Persistent obstructive airways disease (an asthma-like condition) is common among World Trade Center exposed firefighters, though rare in this population before September 11, 2001. (cdc.gov)
  • When it comes to the most common eye color around the world, brown eyes take the cake. (rd.com)
  • They show great commitment to the rare disease community, whose support is key. (mckinsey.com)
  • These pillars might be in place for a few launch archetypes, such as specialized oncology drugs, but the level of commitment and the tactics and capabilities needed to launch a rare disease treatment are of a different order. (mckinsey.com)
  • Access to accurate information about rare disease is inconsistent and infrequent for patients and clinicians. (prnewswire.com)
  • RareU" will align and exponentially increase the availability of educational resources for patients, caregivers, advocates, clinicians, and organizations in the rare disease community. (prnewswire.com)
  • Between 2000 and 2021, more than 200 new orphan medicines were approved by the EMA, addressing the needs of up to 6.3 million rare disease patients. (europabio.org)
  • By harnessing the invaluable experience of the combined organisation in over fifty rare disease indications, we will provide greater value to patients, investigators, and clients. (ddw-online.com)
  • In what parts of the world am I more likely to get onchocerciasis? (cdc.gov)
  • These are airline routes back in 2012 which indicate the different connections of flights between different parts of the world. (cdc.gov)
  • Michael moved to Bordeaux in 2005 and since then has been authenticating private and commercial wine collections around the world. (citizen.co.za)
  • Dallas, TX, September 28, 2011 --( PR.com )-- The Rare Collections is pleased to present the world premier of The Raffaelli Archives Fine Art Prints with an exhibition and appearance by legendary rock photographer Ron Raffaelli on Saturday, October 15 from 12 noon to 2pm at The Rare Collections booth at Guitarlington 2011, the world's largest consumer guitar event in Arlington, Texas. (pr.com)
  • This is an important event in Rock Music History," said Andrew Kirby, Executive Producer of The Rare Collections. (pr.com)
  • The Rare Collections' booth will also feature authentic memorabilia including handwritten poetry by Jim Morrison of The Doors, Tour-worn clothing from Van Halen, fine art photography from Neil Zlozower, vintage guitars, and collectibles from The Beatles, The Rolling Stones, Led Zeppelin, and many more of the best-known and respected artists in the world. (pr.com)
  • The Rare Collections is where top collectors turn to archive, preserve, appraise and disperse their most prized pieces of pop culture and rock-n-roll memorabilia. (pr.com)
  • The Rare Collections booth will be open for the entire event, but Ron Raffaelli will be there from 12 noon to 2pm on Saturday October 15th only. (pr.com)
  • Rohr, a former Colombian Jewish real estate mogul who affiliates as modern Orthodox, and his family have given tens of millions of dollars to Chabad to establish outposts throughout the former Soviet Union, on college campuses and at remote spots around the world. (jta.org)
  • It is very rare and extremely unfortunate that this family had this particular mutation. (sky.com)
  • A rare chemical element, that it is widely used in devices such as smartphones and computers, windscreen glass and self-dimming windows. (edu.au)
  • A (H1N1) circulated widely throughout the world. (cdc.gov)
  • Rare disease affects 1 in 10 people in the US, and 350 million worldwide. (prnewswire.com)
  • Until now, these devices have typically relied on materials like rare indium to do the job. (edu.au)
  • In order to improve the rare disease ecosystem in Europe, policy solutions need to be devised all along the lifecycle of medicines, from screening to patient access. (europabio.org)
  • To be successful, the launch of a rare disease treatment needs a different approach from the standard launch framework. (mckinsey.com)
  • The second-rarest eye color is hazel, a mixture of brown and green with golden flecks. (rd.com)
  • At the same time, the green line indicates the world population. (cdc.gov)
  • In fact, no two people in the world have the same color eyes. (rd.com)
  • The red line indicates the time it took for people to circumnavigate the world. (cdc.gov)
  • But there are also a number of people who travel to other destinations around the world. (cdc.gov)
  • We are excited to increase our impact within the rare disease space, especially as we broaden and deepen our service offerings through the new Emmes rare disease centre. (ddw-online.com)
  • Join more than 1 million activists involved in more than 10,000 projects throughout 160 countries around the world, working to provide good, clean and fair food for all. (slowfood.com)
  • If Moonshot is to succeed, Europe needs a solid pro-innovation ecosystem, supported by a stable intellectual property framework and a world-class regulatory system. (europabio.org)