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PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC`s ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need.
Founded in 2011, PANTHERx Rare is a specialty pharmacy solely focused on rare and orphan diseases, distributing several orphan products and providing access and support services to those needing them most. Known as The Rare Disease Pharmacy®, PANTHERx transforms lives by delivering therapeutic breakthroughs, clinical excellence, personalized support, and a range of access solutions to people living with rare and devastating conditions. While each rare condition affects few people, together all rare diseases impact an estimated 25 to 30 million Americans. Currently over 7,000 rare diseases have been identified and more than 90% of rare diseases are still without an FDA-approved treatment. Changes in federal policy and advances in science have led to a surge in FDA orphan drug approvals, providing tremendous hope to the rare disease community. PANTHERx is a dual-accredited specialty pharmacy, holding distinctions in rare disease including the Accreditation Commission for Health Care (ACHC) Distinction in Orphan Drugs and Utilization Review Accreditation Commission (URAC) Rare Disease Center of Excellence. As a pharmacy focused on patient satisfaction, PANTHERx is the seven-time winner of the prestigious MMIT Patient Choice Award, including the 2024 honor. PANTHERx is headquartered in Pittsburgh, Pennsylvania and is licensed in all 50 states and US territories. #ReinventingSpecialty #RevolutionizingPharmacy #RedefiningCare #DefiningRarePharmacy #TheRareDiseasePharmacy
Caladrius is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of cell therapies designed to reverse disease and/or promote the regeneration of damaged tissue. The Company is developing first-in-class therapeutics based on the characteristics of naturally occurring CD34+ cells and their ability to stimulate the growth of new microvasculature. The Company`s platform technology leverages these cells to enable the body`s natural repair mechanisms using formulations unique to each medical indication.
Lumos Pharma, based in Austin, Texas, is an early stage biotechnology company created to develop and commercialize a novel treatment for the rare disease Creatine Transporter Deficiency. Lumos Pharma is the exclusive licensee of technology and discoveries made in laboratories at the University of Cincinnati and has partnered with Key Opinion Leaders in the field and the United States National Institutes of Health to ensure success in developing a treatment for Creatine Transporter Deficiency.
BioVeris Corporation (BioVeris) is a global healthcare and biosecurity company developing technologies in diagnostics and vaccinology. It develops, manufactures and markets its M-SERIES family of products, which can serve as a platform for diagnostic