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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
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.
At Cradle Genomics, our mission is to provide knowledge that helps every pregnancy.
Poseida Therapeutics is a clinical-stage biotechnology company translating best-in-class technology into lifesaving cell and gene therapies for patients with high unmet medical need. The company is developing CAR T-cell immunotherapies for multiple myeloma, prostate and other cancer types, as well as in vivo gene therapies for orphan genetic diseases. These cell therapies and the gene therapies soon to come are products of our proprietary suite of industry-leading gene engineering technologies, including the piggyBac® DNA Modification System, Cas-CLOVER™ and TAL-CLOVER™ site-specific nucleases.
Morphotek is a Exton, PA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.