https://www.prnewswire.com/news-releases/pegrizeprument-granted-orphan-drug-designation-for-prevention-of-organ-rejection-in-patients-receiving-a-liver-transplant-302666801.html
/PRNewswire/ -- Veloxis Pharmaceuticals, Inc. (Veloxis), a global specialty pharmaceutical company part of Asahi Kasei Corporation, announced today that...
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https://www.technologynetworks.com/genomics/news/stem-cell-based-treatment-for-spinal-muscular-atrophy-receives-orphan-drug-designation-210136
CSC and FSMA reach significant milestone in development of motor neuron treatment for SMA.
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https://www.terrapinn.com/conference/world-orphan-drug-congress-usa/partner-touchendocrinology.stm
touchENDOCRINOLOGY, partnered with - World Orphan Drug Congress USA is the globally leading event for advancing orphan drug development, market access, and...
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https://pubmed.ncbi.nlm.nih.gov/37375769/
Adenylosuccinic acid (ASA) is an orphan drug that was once investigated for clinical application in Duchenne muscular dystrophy (DMD). Endogenous ASA...
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https://www.terrapinn.com/conference/world-orphan-drug-congress-usa/exhibitor-donor-x.stm
Donor X, exhibiting at - World Orphan Drug Congress USA is the globally leading event for advancing orphan drug development, market access, and rare disease...
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https://www.law360.com/healthcare-authority/articles/1835789/pharma-cos-fda-debate-same-drug-in-orphan-drug-case
Two pharmaceutical companies and the U.S. Food and Drug Administration faced off in D.C. federal court Friday over allegations that the federal agency wrongly...
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https://www.biospace.com/press-releases/seed-receives-fda-rare-pediatric-disease-and-orphan-drug-designations-for-lead-oncology-asset-rbm39-degrader-and-enters-strategic-transactions-with-new-investors
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https://www.terrapinn.com/conference/world-orphan-drug-congress-usa/speaker-robert-LONG.stm
Robert Long - World Orphan Drug Congress USA is the globally leading event for advancing orphan drug development, market access, and rare disease patient care.
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https://340borphandrugsolutions.com/
Get official 340B orphan drug solutions designed to simplify orphan drug compliance and support covered entities. Ensure accuracy, avoid penalties, and stay...
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https://www.prnewswire.com/news-releases/bayers-investigational-drug-riociguat-granted-fda-orphan-drug-designation-for-treatment-of-pulmonary-arterial-hypertension-and-chronic-thromboembolic-pulmonary-hypertension-225174802.html
/PRNewswire/ -- Bayer HealthCare today announced that the U.S. Food and Drug Administration's (FDA) Office of Orphan Products Development has granted two...
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https://www.prnewswire.com/news-releases/aridis-pharmaceuticals-receives-orphan-drug-designation-for-ar-501-300875809.html
/PRNewswire/ -- Aridis Pharmaceuticals, Inc. (Nasdaq: ARDS), a biopharmaceutical company focused on the discovery and development of targeted immunotherapies...
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https://www.fiercebiotech.com/biotech/retrophin-buys-up-orphan-technologies-spending-90m-rare-disease-drug
Retrophin has snapped up Orphan Technologies for a $90 million upfront payment, gaining access to the biotech's rare metabolic disorder drug OT-58.
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https://www.terrapinn.com/conference/world-orphan-drug-congress/index.stm?utm_source=partner&utm_medium=touchhaematology&utm_campaign=part&utm_content=homepage&trc=part
The world's largest commercially-focused event for the rare disease industry
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https://www.terrapinn.com/conference/world-orphan-drug-congress-usa/speaker-karen-KAHN.stm
Karen Kahn - World Orphan Drug Congress USA is the globally leading event for advancing orphan drug development, market access, and rare disease patient care.
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