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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
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
The French Muscular Dystrophy Association (AFM-Téléthon)
AFM-Téléthon is an association composed of patients and their families who are affected by a genetic, rare, progressive and severely disabling disease
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.
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Vascular EDS is a life limiting rare genetic disorder
UK registered charity supporting patients & families touched by Vascular Ehlers-Danlos syndrome. Vascular EDS is a rare genetic disorder and is life limiting.
Catabasis
Our mission is to bring hope and life-changing therapies to patients and their families affected by rare diseases, and our primary focus is Duchenne muscular dystrophy.
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Passage Bio, a genetic medicines company, is changing the future of monogenic CNS disease and transforming the lives of patients and their families.
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.
HCB Health—We Give Life-Changing Medicine A Voice
HCB Health is a full-service marketing agency focused on giving life changing medicines a voice. Learn how we can help your novel treatment or therapy
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 […]
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.
SurgingLife | How To Change Your Life, Personal Development For A Surging Life
How To Change Your Life, Personal Development For A Surging Life
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.
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Understanding the world of genetics It doesn’t have to be complicated. It can be as simple as based on a DNA sample to evaluate if your family’s medical history could expose you to a higher-than-usual chance to develop certain diseases like cancer, or to find out how medicines will work on you,
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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 […]
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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.
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