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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
Pharma Conference London | Orphan Drugs & Rare Diseases 2022
Orphan Drugs & Rare Diseases Global Congress is a unique tri-annual Congress for the orphan **** & industry, taking place on the 13th-15th of September 2021.
Author: Paradigm Global Events
Rare Diseases International
Rare Diseases International is the global alliance of people living with a rare disease of all nationalities across all rare diseases
AcceleRare – Advancing **** discovery for rare diseases
Advancing **** discovery for rare diseases
AI Therapeutics - Life Saving Drugs for Rare Diseases
At AI Therapeutics, we are focused on identifying and developing treatments for rare orphan conditions. Our current clinical stage **** candidates target several orphan disorders including pulmonary sarcoidosis, bronchiolitis obliterans post lung transplant and amyotrophic lateral sclerosis (ALS).
Institute for Rare and Neglected Diseases **** Discovery - iRND3
iRND3 is a non-profit company addressing unmet medical needs for rare and neglected diseases by discovering innovative **** molecules for development as affordable, quality, medicines.
Orphan **** Discovery and Development
Orphan **** Discovery and Development
Author: Gennome com
Orphan Disease Therapy and **** Accelerator | Cydan
Cydan accelerates compelling science and develops that path for efficiently launching companies to bring those assets to orphan disease patients. Read more.
Cambridge Rare Disease Network - Cambridge Rare Disease Network
Cambridge Rare Disease Network - Cambridge Rare Disease Network
Author: Suzanne Morris
AllStripes - Be part of the solution for your rare disease
We empower patients and communities to change the way that **** development happens in rare disease.
Orphanet: Emergency guidelines
The portal for rare diseases and orphan drugs
Author: INSERM US
The International Society for Infectious Diseases (ISID) - ISID
The International Society for Infectious Diseases (ISID) brings together a network of over 80,000 individuals from around the world.
Orphanews - Rare diseases newsletter - OrphaNews - International
Orphanews is the Newsletter of the Rare Diseases Community
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...
Rare Disease News, Perspectives & Resources - Rare Disease Advisor
more news >> more features >> Rare Care Podcast Browse Rare Disease Advisor's engaging "Rare Care" podcast series, featuring
Author: Ryner Lai; MBBS
The International Society for the Study of **** Policy (ISSDP) – The International Society for the Study of **** Policy
The International Society for the Study of **** Policy
Amir health care | Drugs and Diseases
Drugs and Diseases
Rare Diseases and Orphan Drugs
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.
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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
Diseasemaps - World Maps of Chronic and Rare Diseases
Connect with people who share your disease and help each other. World Maps of Chronic and Rare Diseases. The main purpose of the project is to help people with chronic and rare diseases. Living with these diseases is usually hard and it is not always properly understood by society. Therefore, we want to spread awareness about all of them and help people who are nowadays suffering. Through this website, you can check how your disease is spread all over the world.
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 ...
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.
Modelis - Simple Models. Complex Diseases. Revolutionizing **** Discovery
Modelis is a world leader in **** discovery for rare and genetic diseases, and is revolutionizing how new therapies are being developed.
BioCryst | **** Drugs for HAE and Other Rare Diseases | Home
BioCryst designs and develops small molecule medicines for rare diseases like hereditary angioedema (HAE) and fibrodysplasia ossificans progressive (FOP).
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.
My Normal | storytelling for rare disease health
My Normal | storytelling for rare disease health
Author: My Normal
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.
#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.
Me, You and PKU – a rare disease, a wonderful life
a rare disease, a wonderful life
Author: Elisakb
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.
Support for patients with chronic illness or rare disease
We provide social-emotional support for patients, teens, young adults, and seniors who are living with chronic illness, rare disease, long-covid, who are undiagnosed, or who are carriers of disease. Connect with patients, make a friend, get support, chronic illness coach, society for teens.
Author: Chronic Warrior Collective
3billion | Genetic test for rare disease patients
Patients with rare genetic disease undergo long odyssey to receive a diagnosis. Visit 3billion and find how to order our rare disease genetic testing.
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.
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
Home | Rare Diseases Clinical Research Network
The Rare Diseases Clinical Research Network (RDCRN), an initiative of the Office of Rare Diseases Research, NCATS, is made up of 21 disease research groups (consortia) and a Data Management and Coordinating Center that work together to improve availability of rare disease information, treatment, clinical studies, and general awareness for both patients and the medical community.
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.
Rare Disease Pharmaceutical Services | Sciensus Rare
We offer bespoke rare disease medicine services; launching & delivering vital medication & clinical services for families that need it most.
UK ATTR Amyloidosis Research Association - Journal of Rare Diseases
Journal of Rare Diseases
Author: Jayden Ortiz
What else icord.es alternative websites
RE(ACT) Congress
The first international congress on rare and orphan diseases, a human platform for knowledge sharing
Crinetics - Developing Therapies For Rare Endocrine Diseases
Crinetics is here for rare endocrine disease patients who are eager to find therapies that provide effective disease control and more simplicity in their lives.
Home | Orphan Resources International
We at Orphan Resources International believe that every child is special and that **** has a plan for each of their lives.
Pharmacenter Ltd. – Orphan **** Market Access Services for Eastern European EU Countries
Pharmacenter Ltd. provides orphan **** market access, marketing & sales as well as packaging services for international pharmaceutical companies in Poland, Hungary, Czech Republic, Slovakia and the Baltic states.
Dolon | Rare diseases strategic pricing and market access consultancy
Treatments for rare diseases face unique challenges in getting to market. Companies developing rare disease treatments need a specialised approach to prepare for pricing and reimbursement negotiations globally. At Dolon, we specialise in strategic pricing and market access for rare and severe diseases.
Khure Health: Identifying Rare Disease Patients
Empowering physicians to identify rare disease patients and put them on the right care pathway.
Author: Drawesome
Rare Disease Foundation
The Rare Disease Foundation is on a mission to find cures and treatments through its innovative patient-centric approach to research. The Foundation also funds and operates programs that provide resources through community and clinical support channels.
CENTOGENE - The Rare Disease Company
CENTOGENE is a world leader in the field of genetic diagnostics for rare diseases dedicated to improve patients' lives every day. Order your genetic test!
Drugs & Health - Side Effects, Disease and Natural Solutions
Drugs and Health for all your disease, drugs and medication side effects. We even discuss natural remedies
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.
Keith McArthur – Author. Podcaster. Speaker. Rare Disease Dad.
Author. Podcaster. Speaker. Rare Disease Dad.
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
Allies in Rare Disease - Global Genes
Global Genes mission is to connect, empower and inspire the rare disease community. Learn more about our events, resources and communities.
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
Allies in Rare Disease - Global Genes
Global Genes mission is to connect, empower and inspire the rare disease community. Learn more about our events, resources and communities.
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
EURenOmics - improving the lives of those with rare kidney disease
EURenOmics aims to predict the disease course, develop novel tools for more accurate diagnoses and new and better therapies for rare kidney diseases.
All God's Children | Adopt An International Orphan - Christian Adoption Agency
All God's Children International is an orphan care ministry dedicated to reaching out in love to the 8 million institutionalized orphans around the world.
Author: Wpengine
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
ERICA | The European Rare Disease Research Coordination and Support Action
ERICA , The European Rare Disease Research Coordination and Support Action
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Cloud-R | Rare disease from a community to data | Italy
Cloud-r digital startup focuses on digital solutions and processes for the collection and analysis of observational data on rare diseases.
Catalyst: A Biopharmaceutical Company Focused on Rare Diseases
Our company is committed to developing novel, safe, and efficacious therapies to improve the lives of people with rare diseases.
Healthcare | Rare Diseases South Africa | Charity
Bridging the gap to improved quality of life for those impacted by Rare Diseases and congenital disorders through advocacy and empowerment.
Cycle Pharmaceuticals – Treatments for rare disease patients
WHAT WE DO Cycle Pharmaceuticals is a global, privately-owned and patient-dedicated biotechnology company headquartered in Cambridge in the UK. Our mission is to utilise the latest pharmaceutical technologies to deliver superior **** treatments to better serve rare genetic disease patients. In addition, we provide individualised patient support services to
Author: Web Admin
Collaborations Pharmaceuticals, Inc.
Our machine learning **** discovery and toxicology capabilities can be applied to rare & neglected disease research and consumer products testing
Healthcare Marketing & Advertising Agency | Dudnyk
Dudnyk is a full-service healthcare marketing and advertising creative agency that specializes in pharmaceutical brand strategy and development for rare diseases and orphan drugs
Peak body for Australians with rare diseases including genetic disorders
Many Voices ONE GOAL YOUR NATIONAL STRATEGIC ACTION PLAN FOR RARE DISEASES Read the Action Plan now Rare Voices Australia (RVA) is the peak national body for Australians living with a rare disease. RVA provides a strong, unified voice to advocate for policy as well as
Rare2Aware – Bringing hope to people with rare diseases
Rare2Aware is a place to learn about rare diseases and bring together patients, their family, caregivers, healthcare professional and advocates
Are you living with a rare disease? | Honeycomb Health
Honeycomb empower patients suffering from rare diseases by providing them with the ability to securely store, access, and share their health information all in one.
Rare Disease Surveillance » Australian Paediatric Surveillance Unit
The Australian Paediatric Surveillance Unit (APSU) conducts national surveillance of rare uncommon diseases in childhood.
CSL Behring | Rare Disease Biotech Company
CSL Behring is a global biotechnology leader driven by its promise to save and improve lives. We manufacture remedies for serious and rare diseases.
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