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Cell and gene therapies have become the third major drug modality in pharmaceutical medicine of the 21st century after low molecular weight and antibody drugs. The gene therapy (GTx) field is rapidly advancing, and yet there are still fundamental scientific questions that remain to be answered. Development of GTx products poses unique challenges and opportunities for drug developers. However, there is lack of a systematic exposition of the GTx product development and the pivotal role of the biostatistician in this process. Development of Gene Therapies: Strategic, Scientific, and Regulatory, and Access Considerations attempts to summarize the current state-of-the-art strategic, scientific, s...
For those who care for chronically ill children, a new understanding of hope that equips adults to better nurture pediatric hope among sick kids—articulated by the children themselves As anyone with a chronic illness knows, hope can sometimes be hard to come by. For parents and caregivers of children with serious illness, there can be a real struggle to move beyond one's own grief, fear, and suffering to see what hope means for these kids. Duane Bidwell, a scholar, minister, and former hospital chaplain who has struggled with serious illness himself, spent time with 48 chronically ill children in dialysis units and transplant clinics around the United States. Chronically ill kids, he found...
Since the inaugural publication of Pediatric Dialysis in 2004, a wide range of advances have taken place in dialysis-related care, leading to a wealth of new knowledge in the field. Pediatric Dialysis, Second Edition brings this knowledge together to provide the most comprehensive source of state-of-the-art information on the dialysis of infants, children and adolescents. With new chapters, updated chapters and references, and contemporary, unique perspectives from authors who are leaders in the global pediatric nephrology community, Pediatric Dialysis, Second Edition is, once again, an authoritative reference that will facilitate best practices in both acute and chronic dialysis. Experienced clinicians and trainees alike will find Pediatric Dialysis, Second Edition not only another valuable contribution to the literature but an indispensable guide to managing their pediatric patients on dialysis.
Written by experts in the field of pharmacovigilance and patient safety, this concise resource provides a succinct, easy-to-digest overview of an increasingly critical area of medical safety. Drs. Thao Doan, Fabio Lievano, Mondira Bhattacharya, and Linda Scarazzini provide essential information for health care professionals, clinical researchers, and regulators who need a comprehensive, up-to-date source of information on the principles and practice of pharmacovigilance. - Covers the evolving regulatory landscape, as well as current and future use of digital technologies. - Uses case studies to ensure content is relevant to everyday practice. - Discusses behavioral science and patient perspectives, risk communication, and new frontiers in pharmacovigilance. - Consolidates today's available information on this timely topic into one convenient resource.
This book covers all key aspects of critical care in pediatric nephrology, including acute dialysis in sick children. It also provides detailed protocols for managing fluid and electrolyte balance and dialysis in children in intensive care. In addition, this quick guide discusses innovations in pediatric renal replacement therapy technologies, such as plasma exchange, CARPEDIEM, NIDUS and aquadex. This is a go-to book for intensivists, physicians and trainees working in pediatric intensive care units.
This new clinical resource brings you a state-of-the-art comprehensive review on every clinical condition encountered in pediatric nephrology in one concise, clinically focused text. International experts provide you with the latest on epidemiology, diagnosis, investigations, management, and prognosis for a full range of pediatric kidney disorders. A full-color, highly visual, meticulously crafted format, makes this material remarkably easy for you to access and apply. Comprehensive Pediatric Nephrology also serves as an ideal resource for board review study for the ABP subspecialty boards in pediatric nephrology. Just the right amount of "need-to-know" basic science coupled with practical c...
This handbook provides the fundamental information required to make good clinical assessments and management decisions in children with renal disorders. It includes topics that range from general pediatric nephrology disorders encountered by primary care physicians to more complex renal disorders managed by pediatric nephrologists. Common pediatric renal conditions including glomerular and tubular disorders, hypertension, and urologic conditions are addressed. Current topics such as neonatal disorders, chronic kidney disease, and transplantation are also covered. In addition, illustrations, algorithms and tables are utilized to highlight teaching points.
Pt. XV. Renal transplantation. ch. 41. Pediatric kidney transplantation : perioperative management / Jens Goebel, William R. DeFoor, Curtis A. Sheldon. ch. 42. Immunosuppressive medications for transplantation / Shefali Mahesh and Deepa H. Chand. ch. 43. Transplant outcomes / Corina Nailescu -- pt. XVI. Cardiovascular disease. ch. 44. Cardiovascular disease in pediatric chronic kidney disease / Amy C. Wilson and Mark M. Mitsnefes -- pt. XVII. Neonatal nephrology. ch. 45. Neonatal renal physiology / Abiodun A. Omoloja. ch. 46. Neonatal disorders / Deepa H. Chand, Heather Stewart and Maria E. de Ferris -- pt. XVIII. Congenital renal diseases. ch. 47. Hydronephrosis, obstructive uropathy, and multicystic dysplastic kidney / Eric Z. Massanyi and Daniel R. McMahon. ch. 48. Polycystic kidney disease / John J. Bissler. ch. 49. Nephronophthisis / Rene G. Van De Voorde III. ch. 50. Rare genetic disorders / Rene G. Van De Voorde III
The new edition of this valuable clinical resource offers a state of the art, comprehensive review on every clinical condition encountered in pediatric nephrology. International experts present the latest knowledge on epidemiology, diagnosis, management, and prognosis in one concise, clinically focused text, in which care has been taken to couple just the right amount of "need-to-know" basic science with practical clinical guidance that will enable the reader to make efficient, informed decisions. The topics covered include: disorders of renal development, glomerular disorders, the kidney and systemic disease, renal tubular disorders, tubulointerstitial disease, urinary tract disorders, acute kidney injury, hypertension, chronic and end-stage renal disease, and renal replacement therapy. The full-color, highly visual, meticulously crafted format will ensure that the practitioner is able to source and apply information with remarkable ease.