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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.
Rare Diseases International
Rare Diseases International is the global alliance of people living with a rare disease of all nationalities across all rare diseases
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.
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.
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.
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!
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.
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 - 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 ...
Verastem Oncology | Biopharmaceutical Company
Verastem Oncology (NASDAQ: VSTM) is a biopharmaceutical company committed to the development of medicines to improve the lives of patients diagnosed with cancer.
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.
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.
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.
Late-Stage Biopharmaceutical Company | Soligenix
Soligenix is a late-stage biopharmaceutical company that develops and commercializes products that treat rare diseases.
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.
Other sites like catalystpharma com
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
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
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.
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.
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
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
Vanda Pharmaceuticals Inc. | A Global Biopharmaceutical Company
Vanda is a global biopharmaceutical company focused on the development and commercialization of innovative therapies to address high unmet medical needs and improve the lives of patients.
Vanda Pharmaceuticals Inc. | A Global Biopharmaceutical Company
Vanda is a global biopharmaceutical company focused on the development and commercialization of innovative therapies to address high unmet medical needs and improve the lives of patients.
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.
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
Melior Pharmaceuticals | Biopharmaceutical Research & Development Company PA
Melior Pharmaceuticals is a mid-stage biopharmaceuticals company in Pennsylvania that develops therapies for diabetes and parkinson’s disease.
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.
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.
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.
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.
RAREVE - relieve people with rare diseases
In Rareve our mission is to relieve people with rare diseases. Build a community, knowledge base and full of support and professional help place for people. If you feel that you need help, or you can help yourself, than…
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.
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.
Global Biopharmaceutical Company - Bristol Myers Squibb
Bristol Myers Squibb is a global biopharmaceutical company committed to discovering, developing and delivering innovative medicines to patients with serious diseases.
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.
What else catalystpharma.com alternative websites
Amryt Pharma Plc - Hope for those with the greatest need
We are a commercial stage pharmaceutical company focused on developing and delivering innovative new treatments that help improve the lives of people with rare and orphan diseases.
Biopharmaceutical Company for Brain Health Disorders - Sage Therapeutics
Sage Therapeutics is a biopharmaceutical company developing novel therapies for brain health disorders with Depression, Neurology and Neuropsychiatric franchise programs.
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
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.
Home - Polyphor
Life-changing therapies: Polyphor’s goal is to be the leader in the development of novel antibiotics and other efficacious therapies against specialty diseases with high medical needs.
Biopharmaceutical company | Pharma Company | Durpha Lifesciences Inc.
We are a biopharmaceutical company focused on development and commercialization of innovative therapies intended to improve outcomes in patient’s sufferings. We are pharma company that prioritizes the needs of the customers and develops superior quality products.
Albireo Pharma, Inc. | Clinical-Stage Biopharmaceuticals
Albireo has a team of diverse specialists who are determined to improve the lives of people living with a wide range of liver diseases. Find out more.
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.
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.
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 […]
Kodiak Sciences - Novel medicines for retinal disease
Our novel therapies for retinal disease are designed to restore vision, stop progression of underlying disease, and require fewer treatments.
Leadiant Biosciences - Research Based Pharmaceautical Company
At Leadiant Biosciences, Inc., our legacy of serving patients is as strong as the future we envision. We developing life-changing therapies for people with rare diseases worldwide.
CSL Behring United Kingdom
CSL Behring is committed to improving the quality of life for people with rare and serious diseases worldwide.
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.
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
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.
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
Mozart Therapeutics | CD8 T regulatory cells | Autoimmune and Inflammatory diseases
Mozart Therapeutics is capitalizing on a novel CD8 T regulatory cell network to develop innovative disease-modifying therapies for autoimmune and inflammatory diseases.
Mendelian: Fixing Diagnosis for Rare Diseases | mendelian.co
Mendelian solutions include the most advanced search engine for Rare Diseases, at home genetic testing, and the latest technology for clinics, and pharma companies.
Author: Mendelian Co
Other websites similar as catalystpharma.com
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.
Khairi Pharmaceuticals
Khairi Pharmaceuticals is committed to finding solutions conducive to the improvement of people's lives with the production, distribution and sale of pharmaceuticals for the treatment of rare, hereditary, degenerative and low-prevalence diseases.
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).
clovisoncology.com | Home
In our pursuit to improve the lives of people living with cancer, Clovis Oncology is committed to realizing the promise of precision medicine for cancer.We seek to develop targeted therapies to better
SYLENTIS S.A. - RNAi biopharmaceuticals
SYLENTIS, biotechnology company focused on the development of new ophthalmic drugs and novel therapies based on RNAi (gene silencig) technology. Addressed diseases in its pipeline includes dry eye, retina diseases, glaucoma or allergies. SYLENTIS is one of the pioneer companies devoted to siRNA, with one of the most advanced siRNA design technologies worldwide, researching on an extense range of Ophtalmical Therapeutic areas and indications. Most advanced experimental drugs: Bamosiran and Tivanisiran.
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.
Oncopeptides
Oncopeptides is a rapidly growing biotech company focused on the development of targeted therapies for difficult-to-treat hematological diseases. The company is science driven and committed to bringing innovation to patients with an unmet medical need and improving patient lives.
Alexion
Alexion is a global biopharmaceutical company focused on developing life-changing therapies for people living with rare disorders.
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.
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
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.
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Laboratorios EDO | Laboratorio veterinario con experiencia y tecnología
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