1. Innovation in treatment for people with rarer or less well-recognised long-term conditions Author(s): Collins, Ben, author Publication: [London, England] : The King's Fund, January 2021 Subject(s): Chronic Disease -- therapyConservative Treatment -- adverse effectsFailure to Rescue, Health CareRare Diseases -- therapySpecializationTime-to-TreatmentInflammatory Bowel Diseases -- therapyOsteoporosis -- therapyPsoriasis -- therapySpondylitis, AnkylosingUnited Kingdom
2. Human gene therapy for rare diseases Publication: Silver Spring, MD : Center for Biologics Evaluation and Research, January 2020 Subject(s): Drug DevelopmentGenetic TherapyRare Diseases -- therapyClinical Trials as TopicDrug Evaluation, PreclinicalUnited StatesUnited States. Department of Health and Human Services.United States. Food and Drug Administration.