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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
Ocugen, Inc. – Pursuing Innovative Ocular Biotherapeutics
Ocugen, Inc. is a clinical stage biopharmaceutical company focused on discovering, developing and commercializing a pipeline of innovative therapies that address rare and underserved eye 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.
MaaT Pharma - We restore microbiome function to treat life-threatening diseases
We are a clinical stage oncology company and industry leader in microbiome restoration therapy using biotherapeutics to treat life-threatening diseases.
Author: MaaT Pharma
Ridgeback Biotherapeutics
Ridgeback Biotherapeutics develops potential medications to treat infectious diseases like Ebola and COVID-19. Learn more.
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.
GeneQuine Biotherapeutics · Home
GeneQuine Biotherapeutics is focused on the development of innovative gene therapy drugs for the treatment of osteoarthritis.
Oyster Point Pharma - Therapies to Address Ophthalmic Disease
We are a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of therapies to address ophthalmic 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.
Microbiota Restoration Therapy™ for Gastrointestinal Diseases | Rebiotix Inc.
A clinical stage microbiome company developing microbiota restoration therapies for the treatment of gastrointestinal diseases from gut dysbiosis.
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.
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.
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 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
Home - Khondrion
Khondrion is a clinical-stage biopharmaceutical company discovering and developing therapies targeting mitochondrial disease.
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).
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
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.
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
Other sites like aeglea com
Home Page - Cancer Prevention
Cancer Prevention Pharmaceuticals is a clinical stage company developing therapeutics to reduce the risk and recurrence of cancer and rare diseases. CPP’s innovative technology addresses polyamine metabolism, immune regulation and inflammation to reduce cancer risk and impact other diseases. These … Continue reading →
Tricida | Working to Turn the Tide on Metabolic Acidosis
Tricida, Inc., is a privately-held, clinical stage, biopharmaceutical company focused on the discovery and development of non-absorbed therapies. Tricida’s lead investigational **** candidate, TRC101, represents a first-in-class approach to the treatment of metabolic acidosis, a common complication of chronic kidney disease (CKD) that can result in increased mortality, accelerated progression of kidney disease, progressive muscle breakdown and exacerbation of bone disease.
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 ...
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.
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.
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.
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.
Personalised Cognitive Behavioral Therapy for Alzheimer's Disease
A Medtech company developing Cognitive Behavioral Therapy (CBT) technologies to address diseases like Alzheimer's Disease, **** etc.
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.
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.
Akero Therapeutics | EFX for NASH | Metabolic Disease | Home
Akero Therapeutics is a cardio-metabolic biotechnology company developing transformational treatments for non-alcoholic steatohepatitis (NASH).
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.
Author: Jpoly4
Home - First Wave BioPharma, Inc.
First Wave BioPharma is a clinical stage biopharmaceutical company specializing in the development of targeted, non-systemic therapies for gastrointestinal (GI) diseases.
Chiasma: Empowering Lives for Everyday Living
Chiasma is a clinical-stage biopharmaceutical company dedicated to addressing the everyday challenges associated with existing therapies for rare or serious chronic diseases.
Athens Research and Technology purified human proteins and enzymes
US manufacturer of purified human proteins, enzymes and cell culture supplements. Our proteins are used in proof of concept studies by researchers developing biotherapeutics. They are also used by researchers looking for biomarkers for cancer and metabolic 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.
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
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.
Chinook Therapeutics | Atrasentan
Chinook is a clinical-stage biopharmaceutical company discovering, developing and commercializing precision medicines for rare, severe chronic kidney diseases.
Arcturus Therapeutics
Arcturus Therapeutics is a clinical-stage messenger RNA medicines company focused on development and distribution of therapeutics for rare diseases & vaccines.
What else aeglea.com alternative websites
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
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.
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 - Aiming to surpass currently available treatments
We are a clinical‑stage biotechnology company striving to deliver novel therapies to patients with neurodegenerative diseases
Caribou Biosciences | Developing Sophisticated Allogeneic Cell Therapies
Caribou is a clinical-stage biopharmaceutical company focused on developing genome-edited allogeneic cell therapies for devastating human 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.
Phenylketonuria Australia, PKU. Maple Syrup **** Disease, MSUD. Inborn errors of metabolism. Low protein diet. Amino acid disorders. | Nutr
Nutrition4me.com.au has been developed to provide extensive information about many aspects of rare inborn errors of metabolism such as phenylketonuria, maple syrup **** disease and fatty acid oxidation disorders.
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).
HemoShear - Discovering New Treatments For Metabolic Disorders | HemoShear Therapeutics
HemoShear is discovering new treatments for rare metabolic disorders. We also partner with biopharma companies to accelerate their **** development.
ProQR Therapeutics - Striving to reverse blindness
ProQR is a clinical stage biotech company that discovers and develops unique RNA therapies for severe genetic disorders, specialized in retinal diseases.
Home - Betagenon
Betagenon is a clinical stage company that develops MPK activators as novel innovative treatments for major diseases.
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.
Natural Metabolics -- Phyteau
Phyteau is developing a new class of incretin-based therapy for diabetes, obesity & related conditions like NASH, NAFLD. The Natural Metabolics are highly effective, safe & side-effect free.
Late-Stage Biopharmaceutical Company | Soligenix
Soligenix is a late-stage biopharmaceutical company that develops and commercializes products that treat 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.
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.
Integrium Clinical Research | Proven leadership in cardiovascular, metabolic disease and dermatology trials | Los Angeles, Tustin, San Diego
Integrium is a full-service Clinical Proof of Concept (PoC) Firm focused within the therapeutic areas of Cardiovascular, Metabolic, Renal, Inflammatory, Dermatology, Wound Healing, Regenerative, and Orphan 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.
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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.
Phathom Pharmaceuticals – Leading the way to innovative treatments for gastrointestinal diseases
A late clinical-stage biopharmaceutical company committed to transforming the treatment landscape for people suffering from GI diseases related to acid.
Home - Amphera
Amphera B.V., a clinical stage Dutch life science company, develops dendritic cell therapies for the treatment of cancer.
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.
Home - Abcuro
Development of immunotherapies for autoimmune diseases and cancer Read more about our approach in Fierce Biotech KLRG1 – A Compelling Clinical Target in Immune Modulation Abcuro is a clinical stage biotechnology company developing first-in-class immunotherapies for autoimmune diseases and cancer … Read More
Author: Abcuro
Home | BioLineRx
BioLineRx is a clinical-stage biopharmaceutical company focused on oncology. The Company in-licenses novel compounds, develops them through pre-clinical and/or clinical stages, and then partners with pharmaceutical companies for advanced clinical development and/or commercialization.
Abcentra - Therapeutics
Abcentra is a clinical-stage biopharmaceutical company developing novel antibody therapeutics for cardiovascular and inflammatory diseases.
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
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 • TC BioPharm
TC BioPharm is a privately-held, clinical-stage cell therapy company developing advanced allogeneic CAR-T cell therapy products for the treatment of cancer.
Home | Algiax Pharmaceuticals GmbH
News – Events About Us Algiax Pharmaceuticals is a clinical-stage biotechnology company established in 2011. It is dedicated to the discovery and development of innovative products to treat diseases with a high unmet medical need. Algiax’ lead candidate AP-325 is a small-molecule GABAA receptor modulator in clinical development as a therapy for neuropathic pain. Next […]
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CTI BioPharma | Targeted Blood Cancer Therapies | Home
CTI BioPharma focuses on the acquisition, development and commercialization of therapies for blood-related cancers that offer unique benefits to patients.
Équilibre Traiteur 🍽 | Service de traiteur à Québec
Pour un service de traiteur à Québec, faites appel à l'équipe d'Équilibre Traiteur. Boîtes à lunch ou événements, nous offrons le meilleur service en ville!
Home - Preiser Scientific
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Vocal Group | Experts in telemarketing and developing your sales teams
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National University Of Health Sciences
National University of Health Sciences offers degrees in chiropractic, naturopathic, oriental medicine, acupuncture, massage therapy, and biomedical science.
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Macrofilter® | Fabricación de filtros de aire, liquido y polvo para la industria
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Experto en la columna vertebral y en su relación íntima con el sistema nervioso. Tratamiento enfocado en neurología, ex consulta del Doctor James Emch.
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Wine Classes and Luxury Wine Tours | Fine Vintage
Fine Vintage offers WSET courses, luxury tours, wine tasting, wine education, specialty courses for consumers, wine lovers and industry professionals.
Individual and Group Health Insurance California | JC Lewis Insurance
We are a family-owned agency specializing in individual and group health insurance in California. Get covered properly with our help!
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Our surf lifesaving patrols Smiths Beach in Yallingup, Western Australia. Get involved as a member, sponsor, or join the Woodside Nippers.
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Nundle | NSW Welcomes You
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Web Design, Printing & Signs in Edinburgh City Centre - Nettl of Edinburgh
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Sage 100, Sage X3, Acumatica ERP - Consulting, Training, Customization
ERP and CRM business software solutions sized for your business from mom-and-pop shops to large enterprises - Sage 100, Sage X3, Acumatica
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NaturEvasion | Activités en plein air au coeur du Var
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Commercial Vehicle Data & Diagnostic Experts | Noregon
Noregon provides commercial vehicle diagnostic & repair software and data analytic solutions for the future of the connected shop and truck.
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