2022
Safety of Deferiprone in Patients with Myelodysplastic Syndromes: Results from the Deferiprone US Safety Registry and a Compassionate Use Program
Zeidan A, Fradette C, Rozova A, Temin N, Tricta F. Safety of Deferiprone in Patients with Myelodysplastic Syndromes: Results from the Deferiprone US Safety Registry and a Compassionate Use Program. Blood 2022, 140: 1115-1117. DOI: 10.1182/blood-2022-169074.Peer-Reviewed Original Research
2018
Availability of Investigational Medicines Through the US Food and Drug Administration’s Expanded Access and Compassionate Use Programs
Puthumana J, Miller JE, Kim J, Ross JS. Availability of Investigational Medicines Through the US Food and Drug Administration’s Expanded Access and Compassionate Use Programs. JAMA Network Open 2018, 1: e180283-e180283. PMID: 30646072, PMCID: PMC6324420, DOI: 10.1001/jamanetworkopen.2018.0283.Peer-Reviewed Original ResearchConceptsCompassionate use programInvestigational medicinesFDA approvalNew drug application submissionAccess programExpanded AccessUS FoodAvailable FDA documentsLife-threatening conditionCross-sectional studyEvidence of safetyProtection of patientsUse programClinical trialsMAIN OUTCOMEDrug AdministrationInfectious diseasesDrug accessClinical development periodsFDA documentsApplication submissionPatientsDiseaseApprovalMonths
This site is protected by hCaptcha and its Privacy Policy and Terms of Service apply