25 published brand profiles in this category. Cite the individual brand profile for brand-specific facts.
Aeglea BioTherapeutics - Aeglea BioTherapeutics is a rare diseases brand headquartered in Austin, TX, founded in 2013.
Amicus Therapeutics - We believe that breakthroughs are possible. That is why we push ideas as far and as fast as possible – in our science, in our work with patients, in everything we do – because every moment matters when you are living with a rare disease. Amicus Therapeutics is a leading global rare-disease focused biotechnology company with a clear and compelling mission: to develop and deliver transformative medicines for people living with rare diseases.
Catabasis Pharmaceuticals - With patients as our guiding stars, Astria Therapeutics’ dedicated and passionate team is devoted to bringing life-changing therapies to patients and families impacted by allergic and immunologic diseases.
Catalyst Pharmaceuticals - People living with rare and difficult-to-treat conditions can’t wait, so we act with urgency, embracing every opportunity to create solutions that help improve someone’s quality of life. That goes beyond developing and commercializing groundbreaking medicines. We partner closely with advocacy groups to enable patients and caregivers, allowing them to make informed decisions that lead to better health outcomes.
Chiesi Farmaceutici - Siamo un gruppo biofarmaceutico orientato alla ricerca che sviluppa e commercializza soluzioni terapeutiche innovative per la salute respiratoria, le malattie rare e le cure specialistiche.
Eiger BioPharmaceuticals - Eiger BioPharmaceuticals is a rare diseases company headquartered in Palo Alto, CA, founded in 2008.
Freeline Therapeutics - Spur Therapeutics is a clinical-stage biotechnology company focused on developing life-changing therapies for debilitating chronic conditions.
Fulcrum Therapeutics - We launched Fulcrum Therapeutics with a bold vision: To change the course of genetically defined diseases by treating them at their root cause.
Global Blood Therapeutics - Global Blood Therapeutics is a rare diseases company headquartered in South San Francisco, CA, founded in 2011.
Homology Medicines - Homology Medicines is a rare diseases company headquartered in Bedford, MA, founded in 2015.
LogicBio Therapeutics - Our Innovation Pushing the Boundaries of Science Our research and development is fueled by a relentless sense of urgency and an steadfast commitment to helping more rare disease patients and their families around the world.Learn how we are addressing some of today’s most pressing rare disease challenges by investigating in new targets, modalities and technologies, with more opportunities for innovation than ever before. To create meaningful advances in rare disease research, it takes an innovati
Orphazyme - We translated this scientific discovery into late-stage clinical trials of our investigational drug candidate, arimoclomol, with the aim of developing new treatment options for people with neurodegenerative diseases, such as Niemann-Pick disease type-C (NPC). In May 2022, substantially all of Orphazyme’s assets and business activities, including those relating to the development and approval of arimoclomol, were sold to KemPharm Denmark A/S, a wholly owned subsidiary of KemPharm, Inc. Orphazyme
Passage Bio - Passage Bio is a rare diseases company headquartered in Philadelphia, PA, founded in 2017.
Poxel - Poxel has two mid-stage products: PXL065 (deuterium-stabilized R-pioglitazone) targeting NASH, which met its primary endpoint in a streamlined Phase 2 trial, and PXL770, a first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator, exclusively focused on rare diseases. Poxel is a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, with a focus on non-alcoholic steatohepatitis (NASH) an
PTC Therapeutics - Therapeutic Areas Diseases in focus Go to previous slide Go to next slide Phenylketonuria (PKU) Metabolic condition caused by mutations in PAH, a key digestive enzyme, that causes cognitive disabilities and seizures Aromatic L-amino acid decarboxylase (AADC) deficiency Patients with AADC deficiency lack dopamine, an essential neurotransmitter that controls movement Duchenne muscular dystrophy (DMD) Rare and fatal genetic disorder resulting in progressive muscle weakness from early childhood Frie
Reata Pharmaceuticals - Reata Pharmaceuticals, Inc. is a pharmaceutical company based in Plano, Texas. Founded in 2002, it is primarily focused on investigating experimental oral antioxidative and anti-inflammatory drugs, which dually activate the antioxidative transcription factor Nrf2 and inhibit the pro-inflammatory transcription factor NF-κB.
Recordati Rare Diseases - The personal data that you provide by compiling this form will be used by Recordati Industria Chimica e Farmaceutica S.p.A.
Retrophin - Travere Therapeutics is a biopharmaceutical company whose mission is to identify, develop and deliver life-changing therapies to people living with rare disease.
Rhythm Pharmaceuticals - Our lead asset, IMCIVREE® (setmelanotide), FDA-approved in 2020, is a precision medicine designed to treat severe obesity caused by an impairment of a pathway in the hypothalamus called the MC4R pathway.
Shire - Takeda is a patient-focused, R&D-driven global biopharmaceutical company committed to bringing Better Health and a Brighter Future.
Sobi - Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Learn more about us here.
Solid Biosciences - Learn more about ways to partner with us in the pursuit of this mission What's New Our Science We have a diversified pipeline across neuromuscular and cardiac diseases. Careers Join our team of experts to advance neuromuscular and cardiac programs. About We are focused on advancing genetic medicines for rare neuromuscular and cardiac 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.
Vivet Therapeutics - VTX-PID is a technology aimed to deplete Neutralizing Antibodies (NAbs) levels for a given AAV serotype, to a level allowing proper AAV transduction and therefore desired efficacy. VTX-PID is developed both for patients presenting NAbs because of a previous infection and therefore non eligible for gene therapy; and for patients in need of gene therapy re-administration in case of loss of efficacy. VTX-806 is an AAV-based gene therapy for Cerebrotendinous xanthomatosis (CTX).
Zynerba Pharmaceuticals - Rooted in empathy for patient needs, we're cultivating growth of innovation that’s essential to advancing the rare neurological disease landscape.