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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.
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.
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.
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.
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!
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
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
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 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.
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.
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.
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.
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
Forma Therapeutics
Forma Therapeutics is a clinical-stage biopharmaceutical company focused on therapeutics to transform the lives of patients with rare hematologic diseases and cancers.
TBTBGLOBAL - Helping the rare and cancer disease communities
We are TBTB Global a company dedicated to help other companies to improve their business and lifestyle of patients dealing with cancer and rare disease.
Sarepta Therapeutics | Biopharmaceutical Company for Rare Diseases
We are focused on the development of precision genetic medicines to treat rare neuromuscular and central nervous system diseases. Learn more about Sarepta.
Sarepta Therapeutics | Biopharmaceutical Company for Rare Diseases
We are focused on the development of precision genetic medicines to treat rare neuromuscular and central nervous system diseases. Learn more about Sarepta.
Sarepta Therapeutics | Biopharmaceutical Company for Rare Diseases
We are focused on the development of precision genetic medicines to treat rare neuromuscular and central nervous system diseases. Learn more about Sarepta.
Other sites like zogenix com
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.
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
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 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.
Impactful Medicines for Rare Neurological Disorders | Ovid Therapeutics
This is Ovid—where we are boldly transforming the lives of people affected by rare neurological diseases including Angelman syndrome, Fragile X syndrome and rare epilepsies.
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.
Cambridge Rare Disease Network - Cambridge Rare Disease Network
Cambridge Rare Disease Network - Cambridge Rare Disease Network
Author: Suzanne Morris
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.
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.
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.
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 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 ...
Travere Therapeutics
Travere Therapeutics is a biopharmaceutical company whose mission is to identify, develop and deliver life-changing therapies to people living with rare disease.
Author: Superadmin
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.
Khure Health: Identifying Rare Disease Patients
Empowering physicians to identify rare disease patients and put them on the right care pathway.
Author: Drawesome
MCDS-Therapy | EU Horizon 2020 project | Rare bone disease
MCDS-Therapy is an EU-funded project. We’re working to develop the first therapy for the rare bone disease, metaphyseal chondrodysplasia type Schmid (MCDS).
Author: Blayne Baker
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.
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.
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
What else zogenix.com alternative websites
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.
Acadia Pharmaceuticals
As a leader in developing and commercializing innovative therapies for central nervous system disorders we are committed to improving the lives of patients.
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
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.
RARE-e-CONNECT - RARE-e-CONNECT
Secure multidisciplinary collaboration and knowledge sharing on rare diseases. For Healthcare Professionals and patients within Cyprus and abroad, large repository of medical centres in Europe and other countries with verified expertise in rare disease groups and rare diseases, Patient life stories, Continuous Medical Education via webinars and automated newsfeeds from key medical databases to stay up to date with important developments and clinical trials in rare diseases.
Author: Super User
ABOUT US | Prilenia Therapeutics
Prilenia Therapeutics is a clinical stage biotech company developing its lead **** candidate, pridopidine, for Huntington’s Disease and ALS. It was founded in 2018 with the purpose of improving the lives of patients and their families by developing treatments for neurodegenerative and neurodevelopmental disorders.
IPM.ai Home Page
IPM.ai transforms RWD via AI to uncover the ideal patient population so pharmaceutical can commercialize therapies for specialty and rare disease
Author: IPM ai
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.
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
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.
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
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.
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.
Alnylam® Pharmaceuticals—The Leading RNAi Therapeutics Company
Alnylam is leading the translation of RNA interference (RNAi) into a innovative, entirely new class of medicines which we believe have the potential to transform the lives of patients who are living with rare and genetic diseases.
Home • Arvinas
Arvinas is a clinical-stage biopharmaceutical company dedicated to improving the lives of patients suffering from debilitating and life-threatening diseases through the discovery, development, and commercialization of therapies that degrade disease-causing proteins.
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.
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.
Eurordis.org
EURORDIS - Rare Diseases Europe, is a non-governmental patient-driven alliance of rare disease patient organisations representing 970 rare diseases patient organisations in 74 countries. We are dedicated to improving the quality of life of all people living with rare diseases in Europe
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
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.
Other websites similar as zogenix.com
Saniona
Improving the lives of rare disease patients around the world through scientific innovation.
Abata Therapeutics
Transformative Therapies for Autoimmune Disease
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.
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.
Author: Wayne Stedman
Zikani Therapeutics
Zikani Therapeutics is a preclinical-stage company developing and commercializing novel therapeutics that improve the lives of patients suffering from serious,life-threatening conditions.
We See Disease Differently | Gemini Therapeutics
Gemini Therapeutics is a precision medicine company focused on the development of new therapies through a deeper understanding of disease.
My Normal | storytelling for rare disease health
My Normal | storytelling for rare disease health
Author: My Normal
PTC Therapeutics
A Science-Led, Patient-Centric Approach to Treating Rare Diseases
PTC Therapeutics
A Science-Led, Patient-Centric Approach to Treating Rare Diseases
PTC Therapeutics
A Science-Led, Patient-Centric Approach to Treating Rare Diseases
PTC Therapeutics
A Science-Led, Patient-Centric Approach to Treating Rare Diseases
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