https://www.dyne-tx.com/
Dyne Therapeutics - A biotechnology company developing nucleic acid therapies for rare diseases.
Mar 5, 2026 - Repairing muscle, reclaiming now. Life-transforming therapies for inherited muscle diseases.
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https://www.eurordis.org/
Home - EURORDIS-Rare Diseases Europe
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https://portal.issn.org/resource/ISSN/2399-5270
ISSN 2399-5270 - Future rare diseases (Online)
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https://www.ukbonn.de/patient_innen/international/english/center-of-rare-diseases/
UKB | Center of Rare Diseases
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https://www.radiorg.be/nl/
Radiorg – Rare Diseases organisation Belgium
Rare Diseases organisation Belgium
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https://www.newstatesman.com/spotlight/reports/2026/02/rare-diseases-closing-the-transition-gap
Rare Diseases: Closing the translation gap - New Statesman
Feb 27, 2026 - A special supplement, sponsored by LifeArc, looking at how Britain can make progress in the battle against rare diseases.
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Rare Diseases Ireland – Supporting rare disease patients and their families in Ireland
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https://irdirc.org/
IRDiRC – International Rare Diseases Research Consortium
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https://www.statnews.com/2026/04/14/hunter-syndrome-avlayah-age-based-approvals/
Age-based approvals for rare diseases can be devastating | STAT
A new therapy for Hunter syndrome highlights the inequities of age-based drug approvals for rare diseases.
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https://www.rarediseasesinternational.org/
Rare Diseases International Rare Diseases International
Apr 13, 2026 - Rare Diseases International is the global alliance of Persons Living with a Rare Disease and their families.
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https://www.rarediseases.co.za/
Healthcare | Rare Diseases South Africa | Charity
Rare Diseases South Africa bridges the gap to improved quality of life for those impacted by Rare Diseases and congenital disorders through advocacy and...
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https://medicover-genetics.com/
Genetic testing for Cancer, Rare diseases & Reproductive health | Medicover Genetics
Mar 31, 2026 - Extensive genetic testing portfolio to help healthcare professionals identify the most appropriate genetic test for the patients. Place genetics at the core of...
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https://www.rare-diseases.eu/
European Conference on Rare Diseases - ECRD2026
Apr 24, 2026 - The ECRD is recognised globally as the largest, patient-led rare disease policy event in which collaborative dialogue, learning and conversation takes place,...
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https://policylabs.frontiersin.org/content/policy-outlook-heumber-perry-bellon-equity-innovation-rare-diseases
Advancing equity through innovation in rare diseases — Frontiers Policy Labs
Apr 14, 2026 - Disruptive approaches such as NANOSPRESSO could help provide equitable access to advanced therapies for patients with rare diseases worldwide, as called for by...
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https://www.newstatesman.com/spotlight/healthcare/2024/10/rare-diseases-innovation-biogen
How we can continue to innovate in the care of rare diseases - New Statesman
Oct 25, 2024 - The UK has a compelling offer in cutting-edge science, but more must be done to realise its potential.
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https://elixir-europe.org/communities/rare-diseases
Rare Diseases Community | ELIXIR
Join this Community The Rare Disease Community extends and generalises the system of access authorisation and high volume secure data transfer developed within...
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https://www.healthing.ca/category/rare-diseases/
Rare Diseases | Healthing.ca
Collectively, rare diseases affect millions, yet patients often face a
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https://www.balgrist.ch/en/offer/specialist-departments/rare-diseases/
Balgrist University Hospital: Rare diseases
[Translate to Englisch:] Anmeldung Sprechstunde: Montag bis Freitag 8.30–12.00 Uhr und 13.00–17.00 Uhr, Tel. +41 44 386 30 95
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https://www.foxnews.com/category/health/medical-research/rare-diseases
Rare Diseases | Fox News
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https://www.cortexbio.co.uk/
CortexBio | Uniting the World of Rare Diseases and Ageing
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https://international.orphanews.org/home.html
Orphanews - Rare diseases newsletter - OrphaNews - International
Orphanews is the Newsletter of the Rare Diseases Community
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https://rarevoices.org.au/
Peak body for Australians with rare diseases including genetic disorders
Feb 15, 2026 - An estimated two million Australians live with a rare disease. Approximately 80% of rare diseases are genetic disorders.
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https://www.genethon.com/
Généthon - Advancing gene therapy treatments for rare diseases
Jan 23, 2026 - Genethon develops innovative therapies for rare diseases of the eye, the liver, the immune system and for neuromuscular diseases.
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https://doaj.org/toc/2399-5270
Future Rare Diseases – DOAJ
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https://www.generetaward.be/
Generet Award for Rare Diseases
Welcome to the Generet Award for Rare Diseases
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https://chiesirarediseases.com/
Chiesi Global Rare Diseases | A Rare Disease Therapeutics Company
Chiesi Global Rare Diseases is a family-owned and run biopharmaceutical company with a commitment to providing transformative care to patients with rare...
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https://www.rarediseases.in/
IORD - Indian Organization for Rare Diseases | A not-for-profit organization for Rare Diseases in...
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https://www.lenovo.com/us/en/case-studies-customer-success-stories/cdfd/
CDFD accelerates research into rare diseases | Lenovo
With data volumes growing, the CDFD established a new high-performance computing cluster powered by Lenovo ThinkSystem and GOAST solutions to help researchers...
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https://www.ciberer.es/en
CIBER Rare Diseases :: Rare Diseases Networking Biomedical Research Centre | CIBERER
Rare Diseases Networking Biomedical Research Centre
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https://ms.cochrane.org/
Home | Cochrane Multiple Sclerosis and Rare Diseases of the CNS
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https://ddrare.nibn.go.jp/
DDrare: Database of Drug Development for Rare Diseases | 難病・希少疾患創薬データベース
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https://www.rd-action.eu/
RD-ACTION : DATA AND POLICIES for rare diseases – RD-ACTION, the new Joint Action consisting of the...
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https://alan.lu/
Rare diseases Luxembourg , we care 4 rare! - ALAN
Jul 25, 2024 - We aim to improve the quality of life of individuals and families affected by rare diseases in Luxembourg. Discover how we can help you.
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https://ordindia.in/
ORD India – Organization for Rare Diseases India
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https://diseasemaps.org/
Diseasemaps - World Maps of Chronic and Rare Diseases
World Maps of Chronic and Rare Diseases and other Syndromes and Conditions - Connect with people who share your condition and help each other
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https://openpolicyfinder.jisc.ac.uk/publication/45064
Future Rare Diseases | Open policy finder
open policy finder provided by Jisc
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https://www.rarediseasesnetwork.org/
Home | Rare Diseases Clinical Research Network
The Rare Diseases Clinical Research Network (RDCRN) is a National Institutes of Health-funded research network working to advance research in rare diseases.
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https://ncats.nih.gov/news-events/news/west-ai-algorithm-may-help-speed-diagnosis-of-rare-diseases
WEST AI Algorithm May Help Speed Diagnosis of Rare Diseases | National Center for Advancing...
NIH-supported researchers trained a new AI algorithm with EHRs to flag rare diseases sooner — may help shorten the diagnostic odyssey.
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https://rarediseasesgreece.com/
RARE DISEASES GREECE
Jan 19, 2026 - Rare Diseases Greece / RDG is a non-profit association based in Athens covering the entire Greek territory, with 28 members – associations, unions, and civic,...
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https://www.menarares.com/
5th MENA Congress for Rare Diseases 2026
5th MENA Congress for Rare Diseases 2026
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https://akadrwill.com/
Will Towler, Ph.D. – A freelance science writer specializing in publications on rare diseases and...
A freelance science writer specializing in publications on rare diseases and skeletal biology, showcasing expertise and portfolio.
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https://www.ppd.com/therapeutic-expertise/oncology-and-hematology-cro/rare-diseases-in-oncology/
Rare Diseases in Oncology and Hematology | PPD
Apr 8, 2026 - The PPD™ clinical research business of Thermo Fisher Scientific advances oncology and hematology rare disease trials through extensive experience,...
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https://recordatirarediseases.com/
Home Page - Recordati Rare Diseases
Mar 30, 2026 - Welcome to Recordati Rare Diseases. We believe that each person with a rare disease has the right to the best treatment
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https://raredisease.nd.edu/
Institute for Rare Diseases | University of Notre Dame
Notre Dame is committed to understanding, treating, and advocating for those affected by rare diseases.
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https://amicusrx.com/
Home - Amicus Therapeutics | Advancing Therapies to Treat Rare Diseases
Apr 27, 2026 - Redefining Expectations in Rare Disease About Amicus Amicus is a wholly owned subsidiary of BioMarin Pharmaceutical Inc., a leading, global rare disease...
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https://www.newstatesman.com/spotlight/2026/03/rare-diseases-from-lived-experience-to-lasting-treatments-sponsored
Rare diseases: from lived experience to lasting treatments - New Statesman
Mar 12, 2026 - Why does diagnosis take so long?
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https://rare2026.com/
The 7th International Conference on Rare Diseases (Rare2026)
Apr 19, 2026 - The 7th International Conference on Rare Diseases (Rare2026), which will take place 17-18 June 2026 in Kraków, Poland.
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https://doaj.org/toc/2399-5270/articles
Future Rare Diseases – DOAJ
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https://solve-rd.eu/
Solve-RD – Solving the Unsolved Rare Diseases
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https://www.sobi.com/en
Sobi - Transforming the lives of people with rare diseases
Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Learn more about us here.
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https://www.ultragenyx.com/
Ultragenyx—Treatment of Rare and Ultrarare Genetic Diseases
Dec 28, 2025 - Ultragenyx is focused on developing first-ever approved treatments for rare and ultrarare diseases and moving the rare disease community forward.
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https://nephcure.org/
NephCure | For Rare Kidney Diseases
Dec 11, 2025 - Founded in 2000, NephCure is the only organization focused on rare kidney diseases, empowering patients and driving research, treatments, and care.
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https://coldagglutininnews.com/news/cell-therapy-helps-woman-rare-combination-3-diseases/
Cell therapy helps woman with rare combination of 3 diseases
Apr 17, 2026 - A CAR T-cell therapy led to rapid, durable easing of symptoms for a woman with severe CAD and two other autoimmune disorders.
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https://pubcasefinder.dbcls.jp/termsofservice
PubCaseFinder: Search system for rare genetic diseases
PubCaseFinder (PCF) is a Human Phenotype Ontology (HPO)-based clinical decision support system for rare and genetic diseases, always up-to-date.
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https://www.sperotherapeutics.com/
Spero Therapeutics | Therapies for Rare & Infectious Diseases
Spero Therapeutics develops innovative treatments for serious rare and infectious diseases, addressing antibiotic resistance and unmet medical needs.
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https://pubcasefinder.dbcls.jp/?lang=en
PubCaseFinder: Search system for rare genetic diseases
PubCaseFinder (PCF) is a Human Phenotype Ontology (HPO)-based clinical decision support system for rare genetic diseases, always up-to-date.
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https://www.capgemini.com/fi-en/news/inside-stories/diagnosing-rare-childhood-diseases/
Diagnosing rare childhood diseases - Capgemini Finland
Dec 3, 2025 - How AI and cloud technology could help speed up medical diagnoses
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https://keck.usc.edu/news/usc-receives-funding-for-ai-tool-to-advance-treatment-for-rare-pediatric-diseases/
USC receives funding for AI tool to advance treatment for rare pediatric diseases
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https://www.capgemini.com/au-en/news/inside-stories/diagnosing-rare-childhood-diseases/
Diagnosing rare childhood diseases - Capgemini Australia
Dec 3, 2025 - How AI and cloud technology could help speed up medical diagnoses
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https://cglife.com/
CG Life | An Agency Specialized for Rare & Hard-to-Treat Diseases
Marketing and communicating the value of new treatments for rare diseases and precision medicines is complex, unpredictable—and deeply personal. CG Life is...
cglifeagencyspecializedrare
https://pubcasefinder.dbcls.jp/
PubCaseFinder: Search system for rare genetic diseases
PubCaseFinder (PCF) is a Human Phenotype Ontology (HPO)-based clinical decision support system for rare genetic diseases, always up-to-date.
genetic diseasessearchsystemrare