Rare DiseasesOrphan Drug ProductionClinical Trials as TopicTreatment OutcomeClinical Trials, Phase III as TopicResearch DesignClinical Trials, Phase II as TopicClinical Trials, Phase I as TopicLymphangioleiomyomatosisDrug ApprovalLithiasisControlled Clinical Trials as TopicDouble-Blind MethodPatient AdvocacyNational Institutes of Health (U.S.)Patient SelectionLegislation, DrugTomography, X-Ray ComputedDrug IndustryUnited States Food and Drug AdministrationActinomycosisPulmonary Alveolar ProteinosisFollow-Up StudiesBiomedical ResearchFatal OutcomeNeuroaxonal DystrophiesTime FactorsPatient RightsRandomized Controlled Trials as TopicHistiocytosis, Langerhans-CellBirt-Hogg-Dube SyndromeHealth Level SevenEuropean UnionBiological OntologiesRetroperitoneal FibrosisUnited StatesHistiocytomaMulticenter Studies as TopicPlacebosClinical Trials Data Monitoring CommitteesUrea Cycle Disorders, InbornAntineoplastic AgentsAcademies and InstitutesErdheim-Chester DiseasePrognosisClinical Trials, Phase IV as TopicClinical ProtocolsEosinophilic GranulomaRetrospective StudiesAngioedemas, HereditaryRegistriesTranslational Medical ResearchCombined Modality TherapyIron Metabolism DisordersMagnetic Resonance ImagingNeoplasmsMedical Laboratory PersonnelProspective StudiesSyndromeSeverity of Illness IndexBreast Neoplasms, MaleEuropeEpidermolysis BullosaSingle-Blind MethodTropherymaOutcome Assessment (Health Care)Early Termination of Clinical TrialsRecurrenceAntineoplastic Combined Chemotherapy ProtocolsRisk FactorsGermanySurvival Analysis