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  1. ramedis.de
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  2. RAMEDIS: Rare Metabolic Diseases Database
    The RAMEDIS system is a platform independent, web-based information system for rare metabolic diseases based on filed case reports
    Author: Bielefeld University Faculty

  3. aeglea.com
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  4. Aeglea BioTherapeutics | Rare Metabolic Disease | Home
    Aeglea BioTherapeutics is a clinical stage company developing enzyme therapies for rare metabolic diseases like Arginase 1 Deficiency and Homocystinuria.

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  6. AZAFAROS – Navigating towards new treatment options for rare metabolic diseases

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  8. Chiesi Global Rare Diseases | Products for rare diseases
    Rare diseases are characterized by a broad diversity of disorders and symptoms that vary not only from disease to disease but also from patient to patient...

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  10. Metabolic Disease Associates | Erie, PA
    Since it's founding in 1973, Metabolic Disease Associates has been Erie’s Choice Specialty Office offering state-of-the-art treatment for Endocrinology, Thyroid, Diabetes & Osteoporosis, and more.

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  12. Cambridge Rare Disease Network - Cambridge Rare Disease Network
    Cambridge Rare Disease Network - Cambridge Rare Disease Network
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  13. rarediseaseadvisor.com
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  14. Rare Disease News, Perspectives & Resources - Rare Disease Advisor
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  15. everylifefoundation.org
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  16. Rare Disease Legislative Advocates | EveryLife Foundation for Rare Diseases

  17. rarediseasefoundation.org
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  18. Rare Disease Foundation
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  20. Rare Disease Database
    Hypermobile Ehlers-Danlos syndrome and physiotherapy were the main keywords of the research. [Results] 1, articles emerged from the.

  21. rarediseasesinternational.org
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  22. Rare Diseases International
    Rare Diseases International is the global alliance of people living with a rare disease of all nationalities across all rare diseases

  23. cureraredisease.org
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  24. Cure Rare Disease
    Cure Rare Disease™ is developing customized therapeutics for those who have been diagnosed with rare, genetic diseases that have no treatment or cures. Through partnerships and collaboration, our world-renowned researchers build life-saving therapeutics that are custom to the patient they are made to treat.

  25. rdi.ie
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  26. Rare Diseases Ireland – Supporting rare disease patients and their families in Ireland

  27. rare-x.org
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  28. Rare Disease Database, Rare Disease Conditions, Federated Data Sharing
    RARE-X, the Rare Disease Database Platform, collects and shares patient-owned health data with researchers across the globe to spur new treatments and cures.

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  29. sciensusrare.com
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  30. Rare Disease Pharmaceutical Services | Sciensus Rare
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  31. cimdrn.ca
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  32. Canadian Inherited Metabolic Diseases Research Network | Canadian Inherited Metabolic Diseases Research Network

  33. rarediseases.in
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  34. IORD - Indian Organization for Rare Diseases | A not-for-profit organization for Rare Diseases in India.

  35. ngocommitteerarediseases.org
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  36. NGO Committee for Rare Diseases
    The NGO Committee for Rare Diseases brings together knowledge and experts to promote rare diseases as a priority in global health, research and social care

  37. centogene.com
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  38. CENTOGENE - The Rare Disease Company
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  39. jrcd.eu
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  40. Journal of Rare Cardiovascular Diseases
    international, quarterly, peer reviewed journal that keeps cardiologists up to date with rare disorders of heart and vessels.
    Author: Podolec P

  41. ultragenyx.com
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  42. Rare Genetic Diseases | Ultragenyx
    Ultragenyx is a clinical-stage biopharmaceutical company committed to bringing to market novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. Founded in 2010, the company has rapidly built a diverse portfolio of product candidates with the potential to address diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no approved therapies.

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  44. HRA Pharma Rare Diseases
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  45. crcd.eu
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  46. Centre for Rare Cardiovascular Diseases
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  47. rarejournal.org
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  48. Rare Diseases and Orphan Drugs

  49. radico.fr
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  50. Radico - Rare Disease Cohorts
    L’objectif principal de RaDiCo est le recueil prospectif, dans le domaine des maladies rares (MR), de données phénotypiques extensives à des fins de recherche clinique et épidémiologique en lien avec la recherche translationnelle et fondamentale. Ces données peuvent être diverses : anatomiques (imagerie médicale), biochimiques, moléculaires, etc.

  51. raredisease.org.uk
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  52. Home - Rare Disease UK
    What's the UK Rare Diseases Strategy? Where can I find information & support? How can I fundraise for Rare Disease UK? The latest news from RARE DISEASE UK... Lauren’s Story Living with Hereditary Amyloidosis One in a Million Abigail’s story Will’s Story Our work We work on ...

  53. mrgm.fr
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  54. Rare Diseases: Genetics and Metabolism – MRGM Research aims to decipher the molecular mechanisms laying at the root of the physiopathology o
    MRGM Research aims to decipher the molecular mechanisms laying at the root of the physiopathology of rare diseases and make our findings worth for patients.

  55. alan.lu
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  56. Rare diseases Luxembourg , we care 4 rare! - ALAN
    We aim to improve the quality of life of individuals and families affected by rare diseases in Luxembourg. Discover how we can help you.

  57. raretraitswiss.ch
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  58. Rare Trait Swiss- AGU Rare Disease: Aspartylglucosaminuria
    Rare Trait Swiss is a Non-Profit organization which contribute to raise awareness of an Lysosomal rare disease called AGU. Our goal is to fund the medical research to manufacture the cure for AGU kids and to fund a clinical trial using gene therapy

  59. scienceofmigraine.com
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  60. Migraine Disease Education for HCPs | Science of Migraine
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  62. BIMDG :: British Inherited Metabolic Disease Group

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  64. There is new hope for the prevention and treatment of metabolic diseases
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  66. Home Page: Nutrition, Metabolism and Cardiovascular Diseases
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  68. CMDGA – Common Metabolic Diseases Genome Atlas

  69. asbmrprimer.com
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  70. Rosen: Primer on the Metabolic Bone Diseases and Disorders of Mineral Metabolism, 8th Edition

  71. diploid.com
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  72. Diploid - Supporting Rare Disease Diagnostics

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  74. F2G - The Rare Fungal Disease Company

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  76. Allies in Rare Disease - Global Genes
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  77. wewearstripes.com
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  78. #WeWearStripes | Rare Disease Day 2021
    We created a rare t-shirt to raise awareness among the general public because rare diseases are not so rare, and for the majority of them there is no cure.

  79. rarediseasesacademy.de
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  80. Startseite - Rare Diseases Academy 2021

  81. ern-lung.eu
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  82. ERN-LUNG | Rare Respiratory Diseases
    WELCOME TO ERN-LUNG ERN-LUNG is a patient-centric network of European healthcare providers and patient organisations, committed Europe-wide and globally to reducing morbidity and mortality from rare lung diseases in people of all ages through patient care, advocacy, education, research and knowledge-sharing. A non-profit project of building a clinical care network for all rare diseases of the respiratory
    Author: Heidi Menges

  83. rare-diseases.eu
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  84. European Conference on Rare Diseases - ECRD2020
    The ECRD is recognised globally as the largest, patient-led rare disease event in which collaborative dialogue, learning and conversation takes place, forming the groundwork to shape future rare disease policies. Leading, inspiring and engaging all stakeholders to take action, the Conference is an unrivalled opportunity to network and exchange invaluable knowledge with all stakeholders in the rare disease community – patient representatives, […]
    Author: Rare Diseases Europe

  85. rarediseaseday.org
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  86. Rare Disease Day ® 2021
    The main objective of Rare Disease Day is to raise awareness with policy makers and the public of rare diseases and of their impact on the lives of patients, and to reinforce their importance as a public health priority.

  87. nirdp.org.uk
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  88. Northern Ireland Rare Disease Partnership

  89. radiorg.be
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  90. Radiorg – Rare Diseases organisation Belgium
    Edelweiss Awards 2021 Belgische koepel voor patiënten met een zeldzame ziekte RaDiOrg is de Belgische koepelvereniging voor mensen met een zeldzame ziekte. Wij tellen meer dan 80 verenigingen voor specifieke zeldzame ziekten en tellen ook honderden individuele leden met een ziekte waarvoor geen vereniging bestaat. Onze missie? Zorgen dat de stem van de zeldzame ziekten wordt gehoord door beleidsmakers. Het grote publiek sensibiliseren voor de zeldzame ziekten. De weg wijzen met heldere informatie. Mensen met zeldzame ziekten verbinden. Ruchtbaarheid geven aan de activiteiten voor en door mensen met zeldzame ziekten. We zijn zeldzaam, maar we zijn talrijk! Nieuws Blijf op

  91. rda-forum.org
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  92. Rare Disease Action Forum | RDAF

  93. zogenix.com
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  94. Zogenix - Therapeutic Solutions for Rare Diseases
    Zogenix is committed to developing and commercializing transformative therapies to improve the lives of patients and their families living with rare diseases.

  95. curecmt4j.org
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  96. CureCMT4J | Advancing Treatments for Rare Diseases

  97. rdodjournal.com
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  98. Rare Disease and Orphan Drugs Journal
    Rare Disease and Orphan Drugs Journal (RDODJ) is an international, peer-reviewed, open access journal. RDODJ will report on scientific advances in the genetics of rare diseases, the molecular basis of the pathologies, and translational research on diagnosis, prevention and treatment. In addition, RDODJ aims to provide a forum for scientific studies and discussion covering the important regulatory, socio-economic and human science issues related to rare diseases and orphan drugs.
    Author: Daniel Scherman

  99. rareve.com
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  100. RAREVE - relieve people with rare diseases
    In Rareve our mission is to relieve people with rare diseases. Build a community, knowledge base and full of support and professional help place for people. If you feel that you need help, or you can help yourself, than…

  101. ordindia.in
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  102. Organization For Rare Diseases India | ORDI
    ORDI was founded to address the many challenges in the management of rare disease in India. A lack of awareness about rare disease even among doctors means that a diagnosis can often take many years.

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  103. mynormal.ca
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  104. My Normal | storytelling for rare disease health
    My Normal | storytelling for rare disease health
    Author: My Normal

  105. rdif.org.in
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  106. Home - Rare Diseases India Foundation

  107. allstripes.com
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  108. AllStripes - Be part of the solution for your rare disease
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  109. globalgenes.org
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  110. Allies in Rare Disease - Global Genes
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  111. rdna.ca
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  112. rDNA | Rare Disease Network of Alberta
    The Rare Disease Network of Alberta (rDNA) is a non-profit corporation that was formed in 2018 to help people understand about rare diseases.

  113. rareopportunities.com
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  114. Welcome to Rare Opportunities in Primary Care, your source for information on primary care in rare diseases

  115. hepatichealth.com
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  116. Liver Disease - HCP Educational Resources | Hepatic Health
    A new educational resource from Gilead Sciences and is designed to meet the educational needs of healthcare professionals in the field of liver disease.

  117. akerotx.com
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  118. Akero Therapeutics | EFX for NASH | Metabolic Disease | Home
    Akero Therapeutics is a cardio-metabolic biotechnology company developing transformational treatments for non-alcoholic steatohepatitis (NASH).

  119. endalldisease.com
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  120. EndAllDisease - Exposing the Metabolic Origins of Disease and How to Reverse It
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  121. takeda-oncology.ch
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  122. Takeda Oncology - for Healthcare Professionals.
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  124. Reflux - for Healthcare Professionals.
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