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  • www.actionduchenne.org
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Hope and Determination on Rare Disease Day 2024 Today, 29th February 2024, marks Rare Disease Day. At Action Duchenne, we join with the global rare disease community to shine a light on the 300 million people across the world living with rare..
Relevance: 35.05973
  • rarediseasesday.wustl.edu
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According to the National Institutes of Health (NIH), a disease is rare if it affects less than 200,000 people in the United States. Over 25 million Americans live with a rare disease-and nearly half of these patients are children. For the over 7,000..
Relevance: 26.731857
  • travere.com
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Travere Therapeutics is a biopharmaceutical company whose mission is to identify, develop and deliver life-changing therapies to people living with rare disease... SAN DIEGO, Feb. 28, 2022 (GLOBE NEWSWIRE) - Travere Therapeutics, Inc. (NASDAQ: TVTX)..
Relevance: 24.591553
  • rdna.ca
The Rare Disease Network of Alberta (rDNA) is a non-profit corporation that was formed in 2018 to help people understand about rare diseases... More than 18,000 Canadians and 2,100 Albertans are expected to have a rare disease and there may be 300..
Relevance: 23.888844
  • kdvsfoundation.org
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The KdVS Foundation is proud to support Rare Disease Day! "Bringing awareness to rare diseases is crucial as it sheds light on the specific challenges faced by those affected. From […]... In honor of Rare Disease Day, we want to share this map..
Relevance: 23.821697
  • www.ultragenyx.com
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Ultragenyx is focused on developing first-ever approved treatments for rare and ultrarare diseases and moving the rare disease community forward... We have built a talented team and a vibrant, people-first culture. We share a passion for helping..
Relevance: 22.731464
  • www.samsaratherapeutics.com
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Samsara Therapeutics, the autophagy biotech company, has appointed eminent neurologist and world-leading researcher in Amyotrophic Lateral Sclerosis (ALS)/Motor Neuron Disease (MND) Professor Dame Pamela Shaw to its scientific advisory board.....
Relevance: 22.355785
  • www.rareuniversity.com
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RARE University is dedicated to providing trusted and relevant education, training, tools, and resources to make an impact in the Rare disease community... All courses are free and designed with you in mind. From basic to complex, Rare University..
Relevance: 22.331686
  • trispera.com
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The world is becoming more and more digitized, so are we! We offer medical website building and newsletter services at present, while many more on the way... We are an Istanbul based pharma solutions company functioning predominantly in Turkey and..
Relevance: 21.705065
  • finding-rare.com
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OUr Team Our team's combined 40+ years in the rare disease marketplace and personal experience with the rare disease diagnostic odyssey led us to believe that there had to be a better way. We knew the data to accelerate rare disease diagnoses and..
Relevance: 21.63729
  • www.winhealthgroups.com
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BO'AO WINHEALTH RARE DISEASE MEDICAL CENTER/BO'AO RARE DISEASE MEDICAL CENTER is keeping pace with development of the world to introduce advanced rare disease medical products into Chinese market, bringing new hope to patients with rare disease in..
Relevance: 21.395256
  • www.sareptatherapeutics.co.uk
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Sarepta is a global biotechnology company on an urgent mission: to engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We're ushering in a new era of drug development with the goal of driving..
Relevance: 21.377052
  • en.sperogenix.com
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Sperogenix Therapeutics is a platform company dedicated to developing and commercializing rare disease therapeutics in China. With prioritized therapeutic areas, such as pulmonary vascular disorders, neuromuscular diseases, and inherited metabolic..
Relevance: 21.26214
  • www.swii.ch
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Swii.ch health is a rare disease patient insight agency and strategy consultancy. We help our healthcare clients make better informed decisions based on a deep understanding of human behaviour... In addition to our core team, we work with a wider..
Relevance: 21.240545
  • www.pscpartnersregistry.org
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The PSC Partners Patient Registry: The registry was created with the guidance of the NIH Office of Rare Disease Research (ORDR). In 2012, PSC Partners was one of the rare disease organizations selected by the ORDR to participate in their Global Rare..
Relevance: 21.025145
  • www.adscientiam.com
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Ad Scientiam is taking this scientific and technological bet, so that today each patient can be an actor in their own health and to accelerate the development of new treatments... On Rare Disease Day, Ad Scientiam, a leader in digital biomarkers,..
Relevance: 20.805307
  • www.squarerootthinking.com
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Square Root is a healthcare consultancy based in Europe. We celebrate the variation in clinical practice, as the hotbed of new emerging best practice and innovation. Adopting simple outcomes as the common currency to codify knowledge and..
Relevance: 20.772722
  • dudnyk.com
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Dudnyk is a full-service healthcare advertising agency that provides rare insights and creativity, with a rare approach, with rare talent, all within the rare disease space...
Relevance: 20.770754
  • thompsoncenter.missouri.edu
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The Thompson Center was inaugurated in 2005 with a generous gift from William and Nancy Thompson (pictured). Based on the medical home model, Thompson Center diagnostic, assessment, and treatment services emphasize family-centered care that is..
Relevance: 20.556995
  • irdirc.org
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The International Rare Diseases Research Consortium (IRDiRC) is a global collaborative initiative launched in 2011 by the European Commission and the US National Institutes of Health to tackle rare diseases through research and accomplish the vision..
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  • www.cureraredisease.org
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Cure Rare Disease, a non-profit organization (Tax ID number 82-2473513), is qualified as a tax-exempt organization under section 501(c)(3) of the IRS and has been designated as a "public charity" under section 170... Cure Rare Disease™ is developing..
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  • topcatbio.com
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Through personal and professional experience in the rare disease community, TOPCAT BIO understands the diagnostic odyssey that the rare disease patient population encounters. TOPCAT BIO also understands the emotional, physical, and fiscal challenges..
Relevance: 20.379095
  • www.blackswanfoundation.ch
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Increasing awareness for rare diseases research and therefore improving the understanding of rare diseases among the public, politicians, health professionals, researchers and academics is one of the main objective of the #RAREvolution. BLACKSWAN..
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  • www.engagehealth.com
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Engage Health is a health research and market development firm who has helped companies in the rare disease space for over 25 years... Engage Health's team provides comprehensive services to further development of rare and specialty disease..
Relevance: 20.246706
  • www.pharming.com
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Pharming is a global biopharmaceutical company developing and commercializing innovative protein replacement therapies and precision medicines to serve the unserved rare disease patient... Pharming's vision is to become the leading global rare..
Relevance: 20.241932
  • www.accredo.com
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Accredo is a specialty pharmacy that serves patients with complex and chronic health conditions, including cancer, hepatitis C, HIV, bleeding disorders and multiple sclerosis. We understand the complexity of these conditions and treatments. Our..
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  • www.rareneuro.com
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Most individuals with a rare disease have never met a specialist in their disorder, and care is inconsistent. Quality of life is often extremely poor, and over 1/3 of deaths in early childhood are due to a rare disease. At the Center for Rare..
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  • connectwithdash.com
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Dash Productions and Event Hosting is an offering from The Dash Alliance for Rare Disease Collaboration, an organization founded in 2015 by rare disease parents and advocates, Eden and Jeff Lord...
Relevance: 19.925802
  • od-expertgroup.eu
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Established in 2020 the European Expert Group on Orphan Drug Incentives (OD Expert Group) brings together representatives of the broad rare diseases community, including researchers, academia, patient representatives, members of the investor..
Relevance: 19.924505
  • oregenetics.com
We believe that stakeholders can mobilize to leverage advances in biotechnology and create a new approach to treating disease, which will make market size increasingly irrelevant. The mission of Oregenetics is to develop a sustainable model for..
Relevance: 19.902819