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La FDA combate las enfermedades raras [[electronic resource] ] : nueva ayuda para pacientes sin tratamientos
| La FDA combate las enfermedades raras [[electronic resource] ] : nueva ayuda para pacientes sin tratamientos |
| Edizione | [Rev. Feb. 2002.] |
| Pubbl/distr/stampa | Rockville, MD : , : Dept. of Health and Human Services, Food and Drug Administration, Office of Public Affairs, , [2002] |
| Descrizione fisica | 1 sheet : digital, PDF file |
| Collana |
Just the facts
Publication |
| Soggetto topico |
Rare diseases - Treatment
Orphan drugs |
| Formato | Materiale a stampa  |
| Livello bibliografico | Monografia |
| Lingua di pubblicazione | spa |
| Altri titoli varianti | FDA combate las enfermedades raras |
| Record Nr. | UNINA-9910695438503321 |
| Rockville, MD : , : Dept. of Health and Human Services, Food and Drug Administration, Office of Public Affairs, , [2002] | ||
| Materiale a stampa | ||
| Lo trovi qui: Univ. Federico II | ||
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FDA fights rare diseases [[electronic resource] ] : new help for patients without treatments
| FDA fights rare diseases [[electronic resource] ] : new help for patients without treatments |
| Edizione | [Rev. Feb. 2002.] |
| Pubbl/distr/stampa | Rockville, MD : , : Dept. of Health and Human Services, Food and Drug Administration, Office of Public Affairs, , [2002] |
| Descrizione fisica | 1 sheet : digital, PDF file |
| Collana |
Just the facts
Publication |
| Soggetto topico |
Rare diseases - Treatment
Orphan drugs |
| Formato | Materiale a stampa |
| Livello bibliografico | Monografia |
| Lingua di pubblicazione | eng |
| Altri titoli varianti | FDA fights rare diseases |
| Record Nr. | UNINA-9910695574303321 |
| Rockville, MD : , : Dept. of Health and Human Services, Food and Drug Administration, Office of Public Affairs, , [2002] | ||
| Materiale a stampa | ||
| Lo trovi qui: Univ. Federico II | ||
| ||
Food and Drug Administration's review process for products to treat rare diseases and neglected tropical diseases [[electronic resource] ] : hearing before a subcommittee of the Committee on Appropriations, United States Senate, One Hundred Eleventh Congress, second session, special hearing, June 23, 2010, Washington, D.C
| Food and Drug Administration's review process for products to treat rare diseases and neglected tropical diseases [[electronic resource] ] : hearing before a subcommittee of the Committee on Appropriations, United States Senate, One Hundred Eleventh Congress, second session, special hearing, June 23, 2010, Washington, D.C |
| Pubbl/distr/stampa | Washington : , : U.S. G.P.O., , 2011 |
| Descrizione fisica | 1 online resource (iii, 58 pages) : illustrations |
| Collana | S. hrg. |
| Soggetto topico |
Rare diseases - Treatment
Tropical medicine Orphan drugs - United States Drug development - Government policy - United States |
| Formato | Materiale a stampa |
| Livello bibliografico | Monografia |
| Lingua di pubblicazione | eng |
| Altri titoli varianti | Food and Drug Administration's review process for products to treat rare diseases and neglected tropical diseases |
| Record Nr. | UNINA-9910703191803321 |
| Washington : , : U.S. G.P.O., , 2011 | ||
| Materiale a stampa | ||
| Lo trovi qui: Univ. Federico II | ||
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Rare diseases : diagnostic and therapeutic odyssey / / edited by Mani T. Valarmathi
| Rare diseases : diagnostic and therapeutic odyssey / / edited by Mani T. Valarmathi |
| Pubbl/distr/stampa | London, England : , : IntechOpen, , [2021] |
| Descrizione fisica | 1 online resource (150 pages) : illustrations |
| Disciplina | 353.00825 |
| Soggetto topico | Rare diseases - Treatment |
| ISBN | 1-83969-412-2 |
| Formato | Materiale a stampa |
| Livello bibliografico | Monografia |
| Lingua di pubblicazione | eng |
| Altri titoli varianti | Rare Diseases |
| Record Nr. | UNINA-9910586681703321 |
| London, England : , : IntechOpen, , [2021] | ||
| Materiale a stampa | ||
| Lo trovi qui: Univ. Federico II | ||
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Rare diseases : expediting treatments for patients : hearing before the Subcommittee on Children and Families of the Committee on Health, Education, Labor, and Pensions, United States Senate, One Hundred Fifteenth Congress, second session, on examining rare diseases, focusing on expediting treatments for patients, October 3, 2018
| Rare diseases : expediting treatments for patients : hearing before the Subcommittee on Children and Families of the Committee on Health, Education, Labor, and Pensions, United States Senate, One Hundred Fifteenth Congress, second session, on examining rare diseases, focusing on expediting treatments for patients, October 3, 2018 |
| Pubbl/distr/stampa | Washington : , : U.S. Government Publishing Office, , 2020 |
| Descrizione fisica | 1 online resource (iii, 40 pages) |
| Collana | S. hrg. |
| Soggetto topico |
Rare diseases - Treatment
Orphan drugs Drug approval |
| Soggetto genere / forma |
Legislative hearings.
Rules. |
| Formato | Materiale a stampa |
| Livello bibliografico | Monografia |
| Lingua di pubblicazione | eng |
| Altri titoli varianti | Rare diseases |
| Record Nr. | UNINA-9910714098603321 |
| Washington : , : U.S. Government Publishing Office, , 2020 | ||
| Materiale a stampa | ||
| Lo trovi qui: Univ. Federico II | ||
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Rare diseases and orphan products [[electronic resource] ] : accelerating research and development / / Committee on Accelerating Rare Diseases Research and Orphan Product Development, Board on Health Sciences Policy ; Marilyn J. Field and Thomas F. Boat, editors ; Institute of Medicine of the National Academies
| Rare diseases and orphan products [[electronic resource] ] : accelerating research and development / / Committee on Accelerating Rare Diseases Research and Orphan Product Development, Board on Health Sciences Policy ; Marilyn J. Field and Thomas F. Boat, editors ; Institute of Medicine of the National Academies |
| Pubbl/distr/stampa | Washington, D.C., : National Academies Press, c2010 |
| Descrizione fisica | 1 online resource (443 p.) |
| Disciplina | 615.19 |
| Altri autori (Persone) |
FieldMarilyn J (Marilyn Jane)
BoatThomas F |
| Soggetto topico |
Orphan drugs
Drugs - Design Rare diseases - Treatment |
| Soggetto genere / forma | Electronic books. |
| ISBN |
1-283-04456-0
9786613044563 0-309-15807-9 |
| Formato | Materiale a stampa |
| Livello bibliografico | Monografia |
| Lingua di pubblicazione | eng |
| Nota di contenuto |
""Front Matter""; ""Acknowledgments""; ""Reviewers""; ""Preface""; ""Contents""; ""Boxes, Figures, and Tables""; ""Summary""; ""1 Introduction""; ""2 Profile of Rare Diseases""; ""3 Regulatory Framework for Drugs for Rare Diseases""; ""4 Discovery Research for Rare Diseases and Orphan Product Development""; ""5 Development of New Therapeutic Drugs and Biologics for Rare Diseases""; ""6 Coverage and Reimbursement: Incentives and Disincentives for Product Development""; ""7 Medical Devices: Research and Development for Rare Diseases""
""8 Toward an Integrated Approach to Accelerating Research and Product Development for Rare Diseases""""References""; ""Appendix A: Study Activities""; ""Appendix B: Innovation and the Orphan Drug Act, 1983-2009: Regulatory and Clinical Characteristics of Approved Orphan Drugs""; ""Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs""; ""Appendix D: Glossary, Abbreviations, and Public Laws""; ""Appendix E: Rare Diseases Clinical Research Network""; ""Appendix F: Advocacy Group Approaches to Accelerating Research and Product Development: Illustrative Examples"" ""Appendix G: Committee and Staff Biographies""""Index"" |
| Record Nr. | UNINA-9910456868603321 |
| Washington, D.C., : National Academies Press, c2010 | ||
| Materiale a stampa | ||
| Lo trovi qui: Univ. Federico II | ||
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Rare diseases and orphan products : accelerating research and development / / Committee on Accelerating Rare Diseases Research and Orphan Product Development, Board on Health Sciences Policy ; Marilyn J. Field and Thomas F. Boat, editors ; Institute of Medicine of the National Academies
| Rare diseases and orphan products : accelerating research and development / / Committee on Accelerating Rare Diseases Research and Orphan Product Development, Board on Health Sciences Policy ; Marilyn J. Field and Thomas F. Boat, editors ; Institute of Medicine of the National Academies |
| Pubbl/distr/stampa | Washington, D.C. : , : National Academies Press, , 2010 |
| Descrizione fisica | 1 online resource (xxi, 420 pages) : illustrations |
| Disciplina | 615.19 |
| Altri autori (Persone) |
FieldMarilyn J (Marilyn Jane)
BoatThomas F |
| Soggetto topico |
Orphan drugs
Drugs - Design Rare diseases - Treatment |
| ISBN |
0-309-20991-9
1-283-04456-0 9786613044563 0-309-15807-9 |
| Formato | Materiale a stampa |
| Livello bibliografico | Monografia |
| Lingua di pubblicazione | eng |
| Nota di contenuto |
""Front Matter""; ""Acknowledgments""; ""Reviewers""; ""Preface""; ""Contents""; ""Boxes, Figures, and Tables""; ""Summary""; ""1 Introduction""; ""2 Profile of Rare Diseases""; ""3 Regulatory Framework for Drugs for Rare Diseases""; ""4 Discovery Research for Rare Diseases and Orphan Product Development""; ""5 Development of New Therapeutic Drugs and Biologics for Rare Diseases""; ""6 Coverage and Reimbursement: Incentives and Disincentives for Product Development""; ""7 Medical Devices: Research and Development for Rare Diseases""
""8 Toward an Integrated Approach to Accelerating Research and Product Development for Rare Diseases""; ""References""; ""Appendix A: Study Activities""; ""Appendix B: Innovation and the Orphan Drug Act, 1983-2009: Regulatory and Clinical Characteristics of Approved Orphan Drugs""; ""Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs""; ""Appendix D: Glossary, Abbreviations, and Public Laws""; ""Appendix E: Rare Diseases Clinical Research Network""; ""Appendix F: Advocacy Group Approaches to Accelerating Research and Product Development: Illustrative Examples""; ""Appendix G: Committee and Staff Biographies""; ""Index"" |
| Record Nr. | UNINA-9910781004803321 |
| Washington, D.C. : , : National Academies Press, , 2010 | ||
| Materiale a stampa | ||
| Lo trovi qui: Univ. Federico II | ||
| ||
Rare diseases and orphan products : accelerating research and development / / Committee on Accelerating Rare Diseases Research and Orphan Product Development, Board on Health Sciences Policy ; Marilyn J. Field and Thomas F. Boat, editors ; Institute of Medicine of the National Academies
| Rare diseases and orphan products : accelerating research and development / / Committee on Accelerating Rare Diseases Research and Orphan Product Development, Board on Health Sciences Policy ; Marilyn J. Field and Thomas F. Boat, editors ; Institute of Medicine of the National Academies |
| Edizione | [1st ed.] |
| Pubbl/distr/stampa | Washington, D.C. : , : National Academies Press, , 2010 |
| Descrizione fisica | 1 online resource (xxi, 420 pages) : illustrations |
| Disciplina | 615.19 |
| Altri autori (Persone) |
FieldMarilyn J (Marilyn Jane)
BoatThomas F |
| Soggetto topico |
Orphan drugs
Drugs - Design Rare diseases - Treatment |
| ISBN |
0-309-20991-9
1-283-04456-0 9786613044563 0-309-15807-9 |
| Formato | Materiale a stampa |
| Livello bibliografico | Monografia |
| Lingua di pubblicazione | eng |
| Nota di contenuto |
""Front Matter""; ""Acknowledgments""; ""Reviewers""; ""Preface""; ""Contents""; ""Boxes, Figures, and Tables""; ""Summary""; ""1 Introduction""; ""2 Profile of Rare Diseases""; ""3 Regulatory Framework for Drugs for Rare Diseases""; ""4 Discovery Research for Rare Diseases and Orphan Product Development""; ""5 Development of New Therapeutic Drugs and Biologics for Rare Diseases""; ""6 Coverage and Reimbursement: Incentives and Disincentives for Product Development""; ""7 Medical Devices: Research and Development for Rare Diseases""
""8 Toward an Integrated Approach to Accelerating Research and Product Development for Rare Diseases""; ""References""; ""Appendix A: Study Activities""; ""Appendix B: Innovation and the Orphan Drug Act, 1983-2009: Regulatory and Clinical Characteristics of Approved Orphan Drugs""; ""Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs""; ""Appendix D: Glossary, Abbreviations, and Public Laws""; ""Appendix E: Rare Diseases Clinical Research Network""; ""Appendix F: Advocacy Group Approaches to Accelerating Research and Product Development: Illustrative Examples""; ""Appendix G: Committee and Staff Biographies""; ""Index"" |
| Record Nr. | UNINA-9910825782703321 |
| Washington, D.C. : , : National Academies Press, , 2010 | ||
| Materiale a stampa | ||
| Lo trovi qui: Univ. Federico II | ||
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Rare Monogenic Diseases : Molecular Pathophysiology and Novel Therapies / / edited by Ivano Condò
| Rare Monogenic Diseases : Molecular Pathophysiology and Novel Therapies / / edited by Ivano Condò |
| Pubbl/distr/stampa | [Place of publication not identified] : , : MDPI - Multidisciplinary Digital Publishing Institute, , 2023 |
| Descrizione fisica | 1 online resource (250 pages) |
| Disciplina | 616.042 |
| Soggetto topico |
Genetic disorders - Treatment
Rare diseases - Treatment |
| ISBN | 3-0365-7460-3 |
| Formato | Materiale a stampa |
| Livello bibliografico | Monografia |
| Lingua di pubblicazione | eng |
| Altri titoli varianti | Rare Monogenic Diseases |
| Record Nr. | UNINA-9910719765703321 |
| [Place of publication not identified] : , : MDPI - Multidisciplinary Digital Publishing Institute, , 2023 | ||
| Materiale a stampa | ||
| Lo trovi qui: Univ. Federico II | ||
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