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Orphagen Pharmaceuticals - Small Molecule Medicine Discovery
Orphagen Pharmaceuticals are leaders in the screening of ligands that modulate orphan nuclear receptors for the discovery of novel medicines
Leading the way to new and better cancer treatments
La Jolla Pharmaceutical Company
La Jolla Pharmaceutical Company is dedicated to the development and commercialization of innovative therapies that improve outcomes in patients suffering from life-threatening diseases
LATITUDE Pharmaceuticals Formulation Experts for Challenging Compounds
A leading contract research specialist, LATITUDE Pharmaceuticals Inc. provides **** formulation services to the biotech and pharmaceutical industries.
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.
we care about rare disease patients | Sobi North America
Sobi is dedicated to developing and delivering innovative therapies to improve the lives of rare disease patients.
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 Diseases International
Rare Diseases International is the global alliance of people living with a rare disease of all nationalities across all rare diseases
Amolyt Pharma - Advancing peptides for the treatment of rare endocrine and metabolic disorders
We are building on our team's established expertise in therapeutic peptides to deliver life-changing treatments to patients suffering from rare endocrine and metabolic diseases.
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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.
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.
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.
Endo-ERN | European Reference Network on Rare Endocrine Conditions
Endo-ERN , European Reference Network on Rare Endocrine Conditions, European Reference Network on Rare Endocrine Conditions. Endo-ERN’s mission is to reduce and ultimately abolish inequalities in care for patients with rare endocrine disorders in Europe, through facilitating knowledge sharing and facilitating related healthcare and research.
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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!
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 ...
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.
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.
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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
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Rare Disease Database, Rare Disease Conditions, Federated Data Sharing
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MCDS-Therapy | EU Horizon 2020 project | Rare bone disease
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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.
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.
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
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.
Khure Health: Identifying Rare Disease Patients
Empowering physicians to identify rare disease patients and put them on the right care pathway.
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Cogent | Precision therapies for genetically defined diseases
Cogent Biosciences is rationally designing precision therapies that treat the underlying cause of disease to improve patients’ lives.
Project Sebastian A Rare Disease Foundation Funding Support
We at Project Sebastian gives support, funding and hope to families affected by a child with a rare disease. Provide education, therapies, and counseling.
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.
Healx | Discovering new treatments for rare disease patients
We're an AI-powered, patient-inspired biotech, and we're breaking new ground in the discovery of new treatments for rare disease patients.
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.
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
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.
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
TBTBGLOBAL - Helping the rare and cancer disease communities
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swii.ch health | Rare Disease Patient Engagement Agency
Focused on rare diseases and specialty markets, we are a patient engagement agency serving pharmaceutical and biotech clients.
Changing the game - The Nathan Project for Rare Diseases
Kids with rare diseases often fall through the cracks of healthcare systems, causing serious problems for patients, their families, and the doctors who treat them. We are developing a needs assessment tool to help doctors and researchers find and fix these gaps, transforming the lives of these families. Left: Our ambassador, Nathan Garcia […]
Rare Trait Swiss- AGU Rare Disease: Aspartylglucosaminuria
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Rare Renal | The UK Kidney Association
It is often hard to find detailed information on rare diseases because they affect so few patients. Doctors may only treat a few patients with a rare disease in their whole career. This site aims to provide information and links to 27 different rare renal conditions and their current activity such as patient support groups and research. Please contact us if you are interested
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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.
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
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Race For 7 | 7000 Rare Diseases | 7000 meters
ORDI team committed to address the challenges of rare disease patients and their families is conducting 'Race for 7', an awareness run in Bengaluru on
Swiss Cell Therapy | Treating Untreatable Diseases
Cell therapy has finally been recognized as a very viable alternative for those diseases with no known therapy or where therapy lacked effectiveness
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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.
DBS Therapy for Parkinson's Disease | Boston Scientific
Deep Brain Stimulation (DBS) has changed the lives of hundreds of thousands of Parkinson's disease patients. Find out now if DBS is right for you.
CSL Behring | Rare Disease Biotech Company
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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
Steve Waugh Foundation | Rare Disease Foundation Australia
Steve Waugh Foundation's mission is to enhance the lives of children and families affected by a rare disease. Grants and fundraising assistance available.
Home - NORD (National Organization for Rare Disorders)
NORD, a 501(c)(3) organization, is the leading patient advocacy organization dedicated to improving the lives of individuals and families living with rare diseases.
Gene Therapy for Rare Disorders Europe 2021 | Home
Dedicated to realising the commerical potential of gene therapies in the rare disease space. Download the full event guide today.
Harmony Biosciences | Patients Are At The Heart Of Everything We Do
Harmony Biosciences is dedicated to developing & commercializing innovative therapies and novel medications for patients living with rare neurological diseases.
Welcome to Eton Pharmaceuticals
Eton is an innovative pharmaceutical company that searches the world over for meaningful therapies that we can bring to patients living with rare diseases.
Add your Lyme disease data to MyLymeData to help find a cure for Lyme disease
MyLymeData is a Lyme disease research project. It lets Lyme disease patients provide data that can help drive research to find a cure for Lyme disease
Welcome to Phage Therapy Center
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Pharmaceutical Company: Medicine Research & Development | Santhera Pharmaceuticals Switzerland
Pharmaceutical company in Switzerland committed to research & development of medicines for patients living with mitochondrial disorders & rare diseases.
Author: X-Ray AG; Switzerland
ERN BOND – European Reference Network on Rare Bone Diseases
Network ERN BOND is one of the 24 existing European Reference Networks (ERNs). In particular, ERN BOND aims to improve access to high-quality healthcare for patients suffering from rare bone diseases. Mission ERN BOND aims to implement measures that facilitate multidisciplinary, holistic and patient-centred care
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Gene and cell therapies to counteract neuroretinal diseases
Find here information on eye disease research and the priority program SPP 2127: Gene and cell based therapies to counteract neuroretinal degeneration.
Personalized Support for Patients & Families Affected by Rare Disease
CareConnectPSS® is designed to support each patient’s unique journey to help you overcome challenges related to living with Fabry, Gaucher, Pompe, and MPS I disease. Learn more about the many support offerings & available resources.
Younique Therapies
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Lung Health Institute | Cellular Therapy for Lung Disease | Breathe Easier™
At the Lung Health Institute, our primary focus is to help patients with chronic lung disease Breathe Easier™. We offer cellular therapy treatments that..
Mito Foundation - Hope for mitochondrial disease
The Mito Foundation drives mitochondrial disease research to find a cure. It also provides support to patients and their families until such time as a cure is found.
Connect Biopharma - Next-Generation Therapeutics for T Cell-Driven Inflammatory Diseases
Connect Biopharma is a global clinical-stage biopharmaceutical company dedicated to improving the lives of patients with chronic inflammatory diseases through the development of therapies derived from T cell-driven research.
Airway Clearance Devices For Chronic Respiratory Diseases | Dymedso
At Dymedso, we are using the power of acoustics to provide effective and gentle airway clearance therapy in a easy-to-use and handheld device for patients.
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Gray Foundation to Cure Batten Disease | Improving the lives of children and families affected by Batten
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Precision Sports Physical Therapy
Precision Sports Physical Therapy provides patients with effective techniques to manage pain, prevent further damage, and achieve optimum function and mobility.
Research | Disease | Sickle Cell Warriors Inc.
Sickle Cell Warriors Inc. provides education, awareness, research access & support for those living with sickle cell disease. We are the largest patient-run sickle cell disease advocacy nonprofit firm in the world, with over 24,000 members. Join the movement to end sickle cell disease. Contact us.
New Paradigm Physical Therapy
Julie Waldo Thieszen and New Paradigm Physical Therapy in Salt Lake City, UT specializes in therapy specific to each individual patient. Highly credentialed, we provide thorough, effective physical therapy to help you recover
Targeted Radioligand Therapy | Advanced Accelerator Applications
Transforming patients’ lives by leading innovation in nuclear medicine and targeted radioligand therapy for oncology indications. Click to find out more.
**** Percussion Therapy for Cystic Fibrosis Patients
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Cartesian | Developing potent, safer cell and gene therapies for cancer and autoimmune diseases | Cartesian
Pioneering RNA-Engineered Cell Therapy In and Beyond Oncology Cartesian uses the RNA Armory®, its proprietary cell therapy platform, to engineer combination therapies into cells, and targets these cells precisely to the site of disease. UNDERSTAND OUR SCIENCE Our therapeutic candidates Cartesian
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Audentes Homepage
Developing TherapiesTo Transform Lives Astellas Gene Therapies is an Astellas Center of Excellence developing genetic medicines with the potential to deliver transformative value for patients. Innovative Therapies Our investigational therapies target rare diseases through three modalities: gene replacement, exon skipping gene therapy and vectorized RNA knockdown.
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PDS Biotech - Home
PDS Biotech is committed to developing novel and effective therapies based on the Versamune® platform to save and improve the lives of cancer patients worldwide. We are also committed to developing safer and more powerful infectious disease vaccines to prevent against selected deadly infectious diseases.
The Ferenc Jolesz National Center for Image Guided Therapy
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Plastic Shop - Tienda de sonido para equipo DJ y Producción Musical
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