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Orphan Drugs
22 Articles Available
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Unmet Needs
In which orphan spaces or unmet medical needs do you think we will see the next treatment innovations?
PAO-06-022--RT-01
Parenterals
Trends Shaping the Dynamic Market for Parenterals
PAP-Q3-20-CL-018
Commercial Manufacturing
Advancing Commercial Manufacturing from Orphan Drugs to Blockbusters
PAP-Q4-19-CL-032
Ophthalmology
Innovative Therapies to Treat Rare and Underserved Eye Diseases
PAP-Q4-19-CL-025
Orphan Drugs
Customer Engagement and Information Sharing are Essential for Success in Orphan Drug Outsourcing
PAP-Q3-19-CL-013
Parenteral Manufacturing
Catering to a Growing Demand for Small-Volume Parenteral Manufacturing
PAO-M04-19-CL-001
Nice Insight Overview
Where Do Orphan Drugs Go From Here?
PAP-Q2-2019-NI-004
Orphan Drug Development
An Expanding Orphan Drug Market
PAP-Q2-2019-NI-001
Orphan Drug Development
Managing the Risks Associated with Orphan Drug Development and Manufacturing
PAP-Q2-2019-NI-002
Orphan Drug Development
Navigating the Road to Successful Orphan Drug Commercialization and Launch
PAP-Q2-2019-NI-003
Clinical Trial Logistics
Facilitating Complex Clinical Trials For Rare Diseases
PAP-Q1-2019-CL-003
Orphan Drug
Cholangiocarcinoma Drug Receives Orphan Drug Designation from FDA
PAO-M03-19-NI-003
FDA
BioInvent Receives FDA Orphan Designation for BI-1206 for Mantle Cell Lymphoma
PR-M01-19-NI-088
Orphan Drug
Arrowhead Receives Positive EMA Opinion on Orphan Designation for ARO-AAT for Treatment of Congenital Alpha-1 Antitrypsin Deficiency
PR-M06-18-NI-097
Orphan Drug
Samus Therapeutics Announces PU-H71 Granted Orphan Drug Designation and First Patient Dosed in Phase 1b Study in Myelofibrosis
PR-M06-18-NI-050
FDA
Scholar Rock Granted Orphan Drug Designation by the FDA for SRK-015 for the Treatment of Patients with Spinal Muscular Atrophy
PR-M04-18-NI-003
Orphan Drug
Kazia Receives FDA Orphan Designation for GDC-0084
PR-M02-18-NI-107
Orphan Drug
Selumetinib Granted Orphan Drug Designation by the U.S. FDA for Neurofibromatosis Type 1
PR-M02-18-NI-76
Orphan Drug Designation
Helsinn Group and MEI Pharma Announce that Pracinostat has Received Orphan Drug Designation from the European Medicines Agency for the Treatment of Acute Myeloid Leukemia (AML)
PR-M01-18-NI-045
FDA
Unjamming the Orphan Drug Backlog
PAO-M07-17-NI-009
FDA Approves Bavencio for Rare Skin Cancer
PAO-M03-17-NI-002
Supporting the Pharma Industry Small Business Growth Engine
PAP-Q1-17-CL-017
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