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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 | Une société biopharmaceutique dédiée aux maladies rares
Nous nous concentrons sur le développement de médicaments génétiques de précision pour traiter les maladies neuromusculaires rares et les maladies du système nerveux central. En savoir plus sur Sarepta.
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.
Anavex Life Sciences
Anavex utilizes precision genetic medicine to treat severe and devastating neurological disorders and is focusing on rare diseases with no available therapy
Samsara Therapeutics - Restoring Autophagy, Reversing Disease
We are restoring autophagy to boost cell rejuvenation and treat currently incurable neurodegenerative and rare genetic 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.
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.
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!
Medicines to Treat Immune-Mediated Diseases | Aldeyra Therapeutics, Inc.
Learn more about Aldeyra Therapeutics, devoted to inventing, developing, and commercializing next-generation medicines that treat immune-mediated disease.
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.
Late-Stage Biopharmaceutical Company | Soligenix
Soligenix is a late-stage biopharmaceutical company that develops and commercializes products that treat rare diseases.
Neurology - Home
Neurosurgery is the surgery discipline focused on treating the central nervous system, peripheral nervous systems and spinal column diseases amenable to surgical intervention.
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.
Convelo Therapeutics
Convelo Therapeutics is a biotechnology company developing a new class of medicines that unlock the regenerative capacity of the central nervous system.
Homepage | Newron Pharmaceuticals
We are a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central and peripheral nervous system
Chinook Therapeutics | Atrasentan
Chinook is a clinical-stage biopharmaceutical company discovering, developing and commercializing precision medicines for rare, severe chronic kidney diseases.
Home - Rhythm Pharmaceuticals
Learn more about how Rhythm is changing how rare genetic diseases of obesity are diagnosed and treated.
Arcturus Therapeutics
Arcturus Therapeutics is a clinical-stage messenger RNA medicines company focused on development and distribution of therapeutics for rare diseases & vaccines.
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Onconova Therapeutics, Inc. - A Biopharmaceutical Company
Onconova Therapeutics, Inc. is focused on discovering and developing novel small molecule **** candidates to treat cancer.
LogicBio Therapeutics - Pioneering Genetic Medicine
LogicBio Therapeutics is a clinical-stage genetic medicine company pioneering gene delivery and gene editing platforms to address rare diseases. Learn More
Neurogene | Developing life-changing Genetic Medicines
Neurogene is focused on developing life-changing genetic medicines for patients & families affected by rare, devastating neurological diseases.
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.
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.
Alnylam® Pharmaceuticals—The Leading RNAi Therapeutics Company
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What is Gaucher disease? Visit GaucherDisease.info
Visit GaucherDisease.info to learn about the rare genetic disorder, Gaucher disease (or Gaucher’s). Symptoms include enlarged liver and spleen, bleeding and bruising, and joint pain.
What is Gaucher disease? Visit GaucherDisease.info
Visit GaucherDisease.info to learn about the rare genetic disorder, Gaucher disease (or Gaucher’s). Symptoms include enlarged liver and spleen, bleeding and bruising, and joint pain.
swii.ch health | Rare Disease Patient Engagement Agency
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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
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Trevena, Inc. (TRVN)
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mRNA Therapeutics for Genetic Disease | Translate Bio
Translate Bio is a clinical-stage mRNA therapeutics company developing a new class of potentially transformative medicines to treat and prevent diseases caused by protein or gene dysfunction.
Minoryx official website
Minoryx is focused on the discovery and development of novel treatments for severe, orphan diseases of the central nervous system with high unmet medical need.
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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.
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.
Multiple Sclerosis (MS) | MS LifeLines®
Learn about multiple sclerosis (MS), a disease that affects the central nervous system, including symptoms, treatment, and community support.
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 - Cognition Therapeutics, Inc.
Treating CentralNervous Systemdisorders by targeting the cellulardamage responsemechanismsTargeting key cellular response mechanisms to fight degenerative diseases. Neurodegenerative diseases like Alzheimer’s disease, Parkinson’s disease, dementia with Lewy bodies and age-related macular degeneration, involve different regions of the central nervous system, yet they share key defects in cellular pathways. By selectively modulating a central cellular damage response … Home Read More »
VectivBio | Transforming Lives
Life-Transforming Medicines for Patients Living with Severe Rare Diseases We are a global, clinical-stage biotechnology company focused on the discovery, development and commercialization of innovative treatments for severe rare conditions with high unmet medical need. Learn More About Us Our Focus We use a patient-centric approach to identify and develop transformative medicines for the treatment […]
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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| Malaysian Rare Disorders Society
Malaysian Rare Disorders Society (MRDS) We represent and look out for the welfare of individuals including their families that are affected by rare disorders. Learn More About MRDS Who We Are Malaysian Rare Disorders Society (MRDS) was formed in 2004, with the help and guidance of the Genetic Unit, Department of Paediatrics, University Malaya Medical […]
Rare Disease Patient Services & Treatment Suite | UltraCare
UltraCare is dedicated to assisting patients in gaining access to Ultragenyx medicines. Learn more about how we can help you. http://UltraCareSupport.com
Neuroimmunology Australia
Neuroimmunology Australia is a collective of neurologists, basic immunologists, neuroscientists and clinical immunologists with an interest in immune-mediated diseases of the central nervous system, the peripheral nerves, the neuromuscular junction and the autonomic nervous system. The aim of Neuroimmunology Australia is to provide a forum for scientific discussion of topics relating to neuroimmunology through our annual workshop, monthly webinars and other media, and to provide learning and training opportunities for young neuroimmunologists.
Functional Medicine | Massachusetts | Newton Integrative Health Services
At Newton Integrative Health Services we practice Functional Medicine which focuses on underlying causes of the disease. Learn more about our practice here.
Altimmune a clinical stage biopharmaceutical company
Altimmune is a clinical stage biopharmaceutical company focused on developing treatments for liver disease and immune modulating therapies. Our diverse pipeline includes treatments for NASH (ALT-801), chronic Hepatitis B, and conjugated immunostimulants for the treatment of cancer. In addition, we are developing intranasal vaccine & therapeutic candidates for anthrax, influenza and COVID-19.
SOCIUM – SOCIUM
SOCIUM is an AI-powered **** discovery and development company focused on first-in-class small molecules. We currently have a development portfolio of over 15 diseases, including oncology and rare diseases. By taking a systems biology approach using our Augmented intelligence and Machine Learning – instead of hypothesizing the mechanism of a disease and focusing on only a few related compounds, we profile the entire disease by analyzing various OMICS data.
Akouos – Our Mission: Healthy Hearing Available to All
Our Mission: Healthy Hearing Available to All. We are a precision genetic medicine company focused on developing gene therapies with the potential to restore, improve and preserve high-acuity physiologic hearing for people worldwide who live with disabling hearing loss.
Author: Ako Admin
Home - Hummingbird
Precision Medicine. Precision Therapies.Hummingbird Bioscience is leading a bold new way of engineering precision biotherapeutics that can define the future of precision medicine. We focus on important biologically validated targets in cancer and autoimmune disease which have been elusive and difficult to ****. Powered by our unique data-driven systems biology approach, we engineer and develop … Continue reading "Home"
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Elvium Life Sciences – Elvium Life Sciences is an innovative life sciences company focused on improving the lives of patients.
About Elvium We are Elvium Life Sciences, a pharmaceutical company passionately committed to improving the health and quality of life of Canadians through innovative medicines in specific therapeutic disease areas of need and importance. Learn More Our Products Elvium Life Sciences focuses on our existing
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Immunotherapy by ImmunityBio - Cell Therapy for Cancer
ImmunityBio is dedicated to conquering cancer. We develop therapies that **** disease, not the immune system. Learn about our investigational immunotherapy and cell therapy products.
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NOCI | Netherlands ****-on-Chip Initiative
In our ageing society, it is more important than ever to develop treatments of devastating chronic diseases. Yet, what appears promising in the lab, often falls short in the clinical practice. We aim to develop a new platform to better predict the effect of medicines, based on a combination of human stem cells and microchips. In this way, we learn much more about the development of diseases and accelerate the discovery of new medicines.
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Home - Viracta Therapeutics, Inc.
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Welcome - Emerald Health Pharmaceuticals
At Emerald Health Pharmaceuticals (EHP), we are focused on the development of a new class of drugs with precise mechanisms of action aimed at bringing innovative therapies to patients to treat diseases with significant unmet medical needs. We believe our novel patented molecules have the potential to be developed into safe and effective pharmaceutical compounds to
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Home - Biosimilars Canada
You can have confidence in your biosimilar medicines Learn moreBiosimilars treat diseases like cancer, diabetes and rheumatoid arthritisLearn moreSwitching to biosimilars is safe and effective for patientsLearn more Previous Next Who we are Biosimilars Canada is a national association representing the biosimilar medicines industry in Canada. We provide leadership in educating Canadian stakeholders about the […]
Keck Medicine of USC
About Keck Medicine of USC – The Keck Effect Keck Medicine of USC is a world-class academic medical center that attracts the world’s top physicians and researchers. Together, we’re preventing, diagnosing and treating diseases with greater precision — an approach we call The Keck Effect. Our world-class medical facilities are staffed by over 500 physicians
NEUROFIT - Validated in vitro and in vivo diseases and safety models for **** screening
NEUROFIT is a preclinical Contract Research Organization specialising in the evaluation of treatments for central and peripheral nervous system disorders. We offer a comprehensive list of in vitro and in vivo models covering a wide range of indications. NEUROFIT aims to facilitate your preclinical **** development by proposing the best models for your needs. Our goal is to accelerate the development of your programs by providing cost-effective methods for compound evaluation.
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IVD, Precision medicine in immunosupression | Biohope
At BIOHOPE we research and develop precision medicine IVD tools for chronic inflammatory conditions. Our lead product, IMMUNOBIOGRAM®, is a novel IVD kit which allows to optimize the immunosuppressive therapy in patients with kidney transplantation and in the future for other autoimmune diseases. THE BIG CHALLENGE CHRONIC INFLAMMATION remains treated with immunosuppressive drugs
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NEUROFIT - Validated in vitro and in vivo diseases and safety models for **** screening - Preclinical Contract Research Organization (CRO) f
NEUROFIT is a preclinical Contract Research Organization specialising in the evaluation of treatments for central and peripheral nervous system disorders. We offer a comprehensive list of in vitro and in vivo models covering a wide range of indications. NEUROFIT aims to facilitate your preclinical **** development by proposing the best models for your needs. Our goal is to accelerate the development of your programs by providing cost-effective methods for compound evaluation.
Author: Bertrand Huyard
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Sarepta Therapeutics | Biopharmazeutisches Unternehmen für seltene Erkrankungen
Wir konzentrieren uns auf die Entwicklung von Präzisionsgenarzneimitteln zur Behandlung seltener neuromuskulärer Erkrankungen und Erkrankungen des zentralen Nervensystems. Erfahren Sie mehr über Sarepta.
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