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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 - Home
PTC Therapuitcs was founded in 1998 and has continuously developed rare disease treatments through innovative science, including **** small molecule drugs and gene therapy.
PTC Therapeutics - Home
PTC Therapuitcs was founded in 1998 and has continuously developed rare disease treatments through innovative science, including **** small molecule drugs and gene therapy.
Home - Caraway Therapeutics
Caraway Therapeutics is pursuing novel approaches for the treatment of debilitating neurodegenerative and rare diseases diseases by enhancing cellular clearance.
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.
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.
Jubilant Therapeutics | Oncology | Autoimmune
As a patient-centric biopharmaceutical company, Jubilant Therapeutics leverages innovation to address unmet medical needs in oncology and autoimmune diseases.
Samsara Therapeutics - Restoring Autophagy, Reversing Disease
We are restoring autophagy to boost cell rejuvenation and treat currently incurable neurodegenerative and rare genetic diseases.
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.
Sutura Therapeutics
At Sutura Therapeutics we are driven to deliver innovative approaches to address current clinical translational bottlenecks in order to positively impact rare and age-related diseases.
Rallybio
Despite science’s best efforts, rare diseases remain far too common. It’s time to tackle the undone, the too difficult, and the inaccessible, and change the odds for patients living with 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.
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
Evolved Science
Evolved Science is a full-service concierge medical practice dedicated to Wellness and Disease Prevention. Evolved Science provides the highest quality medical care in a patient centric environment. We practice our care in an exquisitely designed, curated and approachable setting that provides unpa
Retina International
Retina International (RI) is a patient-led, global, umbrella NGO for patient-led groups around the world focused on research and support for rare and common retinal diseases.
Other sites like ptcbio-en se
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 […]
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 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
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...
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
LightSculpt
Our personalised patient-centric approach is founded in science and backed by research and development – using the latest in preventive ageing and restorative technology.
Author: Solidwebmaster
Periodic Fevers – autoinflammatory conditions
Support, resources for anyone affected by rare autoinflammatory disease; science, articles, patient stories, expert opinion, centre of excellence locator
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.
Home - Better Therapeutics
Pioneering a prescription digital therapeutics platform for treating a range of cardiometabolic diseases.
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.
Arcturus Therapeutics
Arcturus Therapeutics is a clinical-stage messenger RNA medicines company focused on development and distribution of therapeutics for rare diseases & vaccines.
Juventas Therapeutics
Juventas Therapeutics is a privately held biotechnology company developing a pipeline of regenerative therapies to treat life–threatening diseases.
Author: A M Design Group
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
Homepage Ipsen - Fondation Ipsen
Rare Diseases Rare but not alone Mission Accelerate rare disease diagnosis There are 7,000 rare diseases affecting 300 million people worldwide. 75% of patients are children. 1 in 2 patients do not have an accurate diagnosis. A quarter of patients wait 4 years to get a diagnosis. Our program brings together world experts to improve … Homepage Ipsen Read More »
Front Page - BensFriends.org
Safe and Supportive Communities for Rare Disease Patients Donate Today
The Isaac Foundation | Love, Laughter, and Hope
Providing support and advocacy for patients and families coping with rare diseases.
Chinook Therapeutics | Atrasentan
Chinook is a clinical-stage biopharmaceutical company discovering, developing and commercializing precision medicines for rare, severe chronic kidney diseases.
Home | Nice Genes - Charitable clothing brand
nice genes is a brand that advocates for science and the rare disease community.
Dr. Josée Boyer, Naturopathic Doctor - home
is a system of primary care that blends modern science with traditional and natural medicine. Natural therapies are chosen for each individual patient to help treat and prevent disease. By focusing...
RareConnect
A safe, easy to use platform where rare disease patients, families and patient organizations can develop online communities and conversations across continents and languages. RareConnect partners with the world's leading rare disease patient groups to offer global online communities allowing people to connect around issues which affect them while living with a rare disease.
What else ptcbio-en.se alternative websites
Viridian Therapeutics, Inc.
Viridian Therapeutics is a biotechnology company advancing new treatments for patients with diseases that are underserved by today’s therapies.
Dr. Hillel Harris Primary MD Lifestyle Center Health Care - Home
Dr. Hillel Harris treats patients in his office at Primary MD Care, located in Delray Beach, Florida. He provides thoughtful and intuitive primary healthcare in a comfortable, community setting. He aims to treat each patient with compassion and integrity. He utilizes the latest approaches to treating illness and managing chronic disease. He promotes an integrative approach towards wellness, using a combination of lifestyle modification and medications.
Author: Rich Benvin
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
Saniona
Improving the lives of rare disease patients around the world through scientific innovation.
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
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.
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.
Clinical Trial Logistics - Marken
The only patient-centric supply chain organization in the life sciences industry.
Author: Daniel Hennessy
Todd Berner | Patient Advocacy
Todd Berner is a healthcare professional with a passion for patient advocacy, particularly when it comes to patient populations with rare diseases.
Author: Former Co-Chair
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.
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.
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.
Late-Stage Biopharmaceutical Company | Soligenix
Soligenix is a late-stage biopharmaceutical company that develops and commercializes products that treat rare diseases.
EUROCAT | EU RD Platform
Providing a central access point for information on rare disease patients’ registries to all stakeholders
Vivoryon Therapeutics - Home
Vivoryon Therapeutics is a precision medicine company focused on bringing first-in-class therapies to patients suffering from age-related diseases.
UroGen Pharma is Making the Impossible Possible
A bold approach to building novel solutions that treat specialty cancers and urologic diseases.
Akcea Therapeutics
About Akcea Learn about Akcea’s core values and hear from Akcea Canada’s Vice President & GM, Jared Rhines. More about our vision & mission Our focus Learn about two rare diseases: FCS and hATTR amyloidosis. More about our approach Our innovation Read about our products and our clinical trials. More about our products Our commitment … Continue reading "Home"
Home - Rhythm Pharmaceuticals
Learn more about how Rhythm is changing how rare genetic diseases of obesity are diagnosed and treated.
The Genesis Foundation For Children | More Than Medical Care
The Genesis Foundation for Children diagnoses, treats and provide coordinated care for children with rare diseases and genetic disorders.
Other websites similar as ptcbio-en.se
Lyme Disease Institute of Texas | San Antonio, TX
Located in San Antonio, Texas, the Lyme Disease Institute of Texas uses an integrative approach to diagnose and treat Lyme disease.
Home Page - Cerevel Therapeutics
Unraveling the mysteries of the brain to treat neuroscience diseases Cerevel Therapeutics is working relentlessly to find paths through complexity in an effort to bring real progress and new treatment options to people living with a broad range of neuroscience 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.
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.
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.
Parathyroid Questions and Answers
Parathyroid Q&A is a community of experts and patients dedicated to understanding and treating Parathyroid Disease.
Egetis Therapeutics
Egetis is an innovative, unique, and integrated pharmaceutical **** development company, focusing on projects in late-stage development for treatment of serious rare/niche diseases with significant unmet medical needs in the orphan **** segment
DiseaseFix - World's Largest Disease Information Center
Leading disease-centric medical information center. Read comprehensive material on diseases to find knowledge at one place | Patient education, Guidelines, Images, Photos, Graphics.
Remix Therapeutics | Reprogramming RNA Processing
Remix Therapeutics is developing small molecule therapies designed to reprogram RNA processing and treat disease in entirely new ways.
Home - Talaris Therapeutics
Talaris seeks to help kidney transplant recipients and patients suffering from severe auto-immune diseases to live healthy lives free from chronic immunosuppression
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
