Rare Diseases and Orphan Products

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Author :
Publisher : National Academies Press
ISBN 13 : 0309158060
Total Pages : 442 pages
Book Rating : 4.3/5 (91 download)

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Book Synopsis Rare Diseases and Orphan Products by : Institute of Medicine

Download or read book Rare Diseases and Orphan Products written by Institute of Medicine and published by National Academies Press. This book was released on 2011-04-03 with total page 442 pages. Available in PDF, EPUB and Kindle. Book excerpt: Rare diseases collectively affect millions of Americans of all ages, but developing drugs and medical devices to prevent, diagnose, and treat these conditions is challenging. The Institute of Medicine (IOM) recommends implementing an integrated national strategy to promote rare diseases research and product development.

Orphan

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Author :
Publisher :
ISBN 13 : 9781621821373
Total Pages : 0 pages
Book Rating : 4.8/5 (213 download)

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Book Synopsis Orphan by : Philip Reilly

Download or read book Orphan written by Philip Reilly and published by . This book was released on 2015 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: "This book is about the struggle to save the lives of children who, because of a roll of the genetic dice, are born with any one of more than several thousand rare genetic disorders. It recounts the now century long effort of small groups of physicians and scientists to take on some of these genetic diseases. In many cases just a few physician-scie

Orphan Drugs and Rare Diseases

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Author :
Publisher : Royal Society of Chemistry
ISBN 13 : 1782624201
Total Pages : 487 pages
Book Rating : 4.7/5 (826 download)

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Book Synopsis Orphan Drugs and Rare Diseases by : David Pryde

Download or read book Orphan Drugs and Rare Diseases written by David Pryde and published by Royal Society of Chemistry. This book was released on 2014-07-30 with total page 487 pages. Available in PDF, EPUB and Kindle. Book excerpt: Orphan drugs are designated drug substances that are intended to treat rare or ‘orphan’ diseases. More than 7000 rare diseases are known that collectively affect some 6-7% of the developed world’s population; however, individually, any single, rare disease may only affect a handful of people making them commercially unattractive for the biopharmaceutical industry to target. Ground breaking legislation, starting with the Orphan Drug Act that was passed in the US in 1983 to provide financial incentives for companies to develop orphan drugs, has sparked ever increasing interest from biopharmaceutical companies to tackle rare diseases. These developments have made rare diseases, and the orphan drugs that treat them, sufficiently attractive to pharmaceutical development and many pharmaceutical companies now have research units dedicated to this area of research. It is therefore timely to review the area of orphan drugs and some of the basic science, drug discovery and regulatory factors that underpin this important, and growing, area of biomedical research. Written by a combination of academic and industry experts working in the field, this text brings together expert authors in the regulatory, drug development, genetics, biochemistry, patient advocacy group, medicinal chemistry and commercial domains to create a unique and timely reference for all biomedical researchers interested in finding out more about orphan drugs and the rare diseases they treat. Providing an up-to-date monograph, this book covers the basic science, drug discovery and regulatory elements behind orphan drugs and will appeal to medicinal and pharmaceutical chemists, biochemists and anyone working within the fields of rare disease research and drug development or pharmaceuticals in industry or academia.

Orphan Justice

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Publisher : B&H Publishing Group
ISBN 13 : 1433677970
Total Pages : 224 pages
Book Rating : 4.4/5 (336 download)

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Book Synopsis Orphan Justice by : Johnny Carr

Download or read book Orphan Justice written by Johnny Carr and published by B&H Publishing Group. This book was released on 2013-03-01 with total page 224 pages. Available in PDF, EPUB and Kindle. Book excerpt: Christians are clearly called to care for orphans, a group so close to the heart of Jesus. In reality, most of the 153 million orphaned and vulnerable children in the world do not need to be adopted, and not everyone needs to become an adoptive parent. However, there are other very important ways to help beyond adoption. Indeed, caring for orphaned and vulnerable children requires us to care about related issues from child trafficking and HIV/AIDS to racism and poverty. Too often, we only discuss or theologize the issues, relegating the responsibility to governments. No one can do everything, but everyone can do something. Based on his own personal journey toward pure religion, Johnny Carr moves readers from talking about global orphan care to actually doing something about it in Orphan Justice. Combining biblical truth with the latest research, this inspiring book: • investigates the orphan care and adoption movement in the U.S. today • examines new data on the needs of orphaned and vulnerable children • connects “liberal issues” together as critical aspects or orphan care • discovers the role of the church worldwide in meeting these needs • develops a tangible, sustainable action plan using worldwide partnerships • fleshes out the why, what, and how of global orphan care • offers practical steps to getting involved and making a difference

The Price of an Orphan

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Author :
Publisher :
ISBN 13 :
Total Pages : 190 pages
Book Rating : 4.:/5 (6 download)

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Book Synopsis The Price of an Orphan by : Patricia Carlon

Download or read book The Price of an Orphan written by Patricia Carlon and published by . This book was released on 1964 with total page 190 pages. Available in PDF, EPUB and Kindle. Book excerpt:

The Orphan

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Author :
Publisher : Fisher King Press
ISBN 13 : 1771690178
Total Pages : 178 pages
Book Rating : 4.7/5 (716 download)

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Book Synopsis The Orphan by : Audrey Punnett

Download or read book The Orphan written by Audrey Punnett and published by Fisher King Press. This book was released on 2014-06-21 with total page 178 pages. Available in PDF, EPUB and Kindle. Book excerpt: The Orphan: A Journey to Wholeness addresses loneliness and the feeling of being alone in the world, two distinct characteristics that mark the life of an orphan. Regardless if we have grown up with or without parents, we are all too likely to meet such experiences in ourselves and in our daily encounters with others. With numerous case examples, Dr. Punnett describes how loneliness and the feeling of being alone tend to be repeated in later relationships and may eventually lead to states of anxiety and depression. The main purpose of this book is not to just stay within the context of the literal orphan, but also to explore its symbolic dimensions in order to provide meaning to the diverse experiences of feeling alone in the world. In accepting the orphan within, we begin to take responsibility for our own unique life journey, a privileged journey in which one can at some point in time say with pride, I am an orphan.

Orphan Train

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Author :
Publisher : HarperLuxe
ISBN 13 : 9780062887870
Total Pages : 432 pages
Book Rating : 4.8/5 (878 download)

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Book Synopsis Orphan Train by : Christina Baker Kline

Download or read book Orphan Train written by Christina Baker Kline and published by HarperLuxe. This book was released on 2019-01-08 with total page 432 pages. Available in PDF, EPUB and Kindle. Book excerpt: From Christina Baker Kline comes a novel about two women: one about to age out of the foster care system, the other 90 years old and carrying both a tremendous secret and a story of a life formed by a part of American history almost entirely forgotten: the Orphan Trains Molly Ayer has one last chance, and she knows it. Close to being kicked out of her foster home -- just months from turning 18 and “aging out” of the system -- Molly should be grateful that her boyfriend found her a community service project: helping an old lady clean out her home. Molly can’t help but think that the 50 hours will be tedious, but at least they’ll keep her out of juvie, and right now that’s all she cares about. Ninety-one-year-old Vivian Daly has lived a quiet life on the coast of Maine for decades. But in her attic, hidden in trunks, are keys to a turbulent past. Molly is about to discover -- as she and Vivian unpack her possessions, and memories -- that Vivian’s story is a piece of America’s tumultuous history now largely forgotten: the tale of a young Irish immigrant, orphaned in New York City and put on a train to the Midwest with hundreds of other orphaned children whose destiny would be determined by luck and chance. As Molly digs deeper, she finds surprising parallels in her own experience as a Penobscot Indian and Vivian’s story -- and Molly realizes that she has the power to help Vivian find answers to mysteries that have haunted her for her entire life. Rich in detail and epic in scope, THE TRAIN RIDER is a powerful novel of upheaval and resilience, of second chances, of unexpected friendships, and of the secrets we carry with us that keep us from finding out who we are.

The Song of the Orphans

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Author :
Publisher : Penguin
ISBN 13 : 0399164995
Total Pages : 753 pages
Book Rating : 4.3/5 (991 download)

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Book Synopsis The Song of the Orphans by : Daniel Price

Download or read book The Song of the Orphans written by Daniel Price and published by Penguin. This book was released on 2017-07-04 with total page 753 pages. Available in PDF, EPUB and Kindle. Book excerpt: The thrilling second novel in the category-defying Silvers trilogy—melding X-Men and the novels of Blake Crouch—about six extraordinary people who become unwitting refugees on an unfamiliar Earth, and their epic quest to find out why. The end of the world was just the beginning for Hannah and Amanda Given. Saved from apocalypse by three mysterious beings, the sisters, along with four other refugees from their world, were each marked with a silver bracelet and transported to an entirely different Earth: a place where restaurants move through the air like flying saucers and the fabric of time is manipulated by common household appliances, as well as by their very own hands—and a place where terrifying new adversaries seem to be around every corner. Now, after six months in this alt-America and a tumultuous cross-country journey that landed them in New York City, the Silvers find themselves in more trouble than ever. Their new world is dying, and a clan of powerful time benders believes that killing them is the only way to stop it. To make matters worse, the U.S. government has sent its most ruthless covert spy agency to track and capture them. But the biggest threat of all comes from the three god-like beings who first saved them. They had a reason for bringing the Givens and their friends to this world. And when the Silvers learn the awful truth, nothing will ever be the same.

Rare Diseases and Orphan Drugs

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Author :
Publisher : Academic Press
ISBN 13 : 0124200095
Total Pages : 407 pages
Book Rating : 4.1/5 (242 download)

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Book Synopsis Rare Diseases and Orphan Drugs by : Jules J. Berman

Download or read book Rare Diseases and Orphan Drugs written by Jules J. Berman and published by Academic Press. This book was released on 2014-05-26 with total page 407 pages. Available in PDF, EPUB and Kindle. Book excerpt: Rare Diseases and Orphan Drugs shows that much of what we now know about common diseases has been achieved by studying rare diseases. It proposes that future advances in the prevention, diagnosis, and treatment of common diseases will come as a consequence of our accelerating progress in the field of rare diseases. Understanding the complex steps in the development of common diseases, such as cancer, cardiovascular disease, and metabolic diseases, has proven a difficult problem. Rare diseases, however, are often caused by aberrations of a single gene. In rare diseases, we may study how specific genetic defects can trigger a series of events that lead to the expression of a particular disease. Often, the disease process manifested in a certain rare disease is strikingly similar to the disease process observed in a common disease. This work ties the lessons learned about rare diseases to our understanding of common ones. Chapters covering the number of common diseases are minimized, while rare diseases are introduced as single diseases or as members of diseases classes. After reading this book, readers will appreciate how further research into the rare diseases may lead to new methods for preventing, diagnosing, and treating all diseases, rare or common. Makes rare diseases relevant to clinicians and researchers by tying lessons learned about the rare diseases to our understanding of the common diseases Stresses basic pathologic mechanisms that account for human disease (e.g., disorders of cell development, replication, maintenance, function and structure), that can be understood without prior training in pathology Discusses advanced concepts in molecular biology and genetics in a simple, functional context appropriate for medical trainees and new researchers Offers insights into how further research into rare diseases may lead to new methods for preventing, diagnosing, and treating all diseases

Midlife Orphan

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Author :
Publisher : Berkley
ISBN 13 :
Total Pages : 250 pages
Book Rating : 4.0/5 ( download)

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Book Synopsis Midlife Orphan by : Jane Brooks

Download or read book Midlife Orphan written by Jane Brooks and published by Berkley. This book was released on 1999-04 with total page 250 pages. Available in PDF, EPUB and Kindle. Book excerpt: This thoughtful exploration of a neglected subject explains the emotional impact of losing parents in the midst of midlife--and why many underestimate it.

The Ogress and the Orphans

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Publisher : Hachette UK
ISBN 13 : 1643752871
Total Pages : 393 pages
Book Rating : 4.6/5 (437 download)

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Book Synopsis The Ogress and the Orphans by : Kelly Barnhill

Download or read book The Ogress and the Orphans written by Kelly Barnhill and published by Hachette UK. This book was released on 2022-03-08 with total page 393 pages. Available in PDF, EPUB and Kindle. Book excerpt: A National Book Award finalist and instant fantasy classic about the power of community, generosity, books, and baked goods, from the author of the beloved Newbery Medal winner The Girl Who Drank the Moon. Stone-in-the-Glen, once a lovely town, has fallen on hard times. Fires, floods, and other calamities have caused the people to lose their library, their school, their park, and even their neighborliness. The people put their faith in the Mayor, a dazzling fellow who promises he alone can help. After all, he is a famous dragon slayer. (At least, no one has seen a dragon in his presence.) Only the clever children of the Orphan House and the kindly Ogress at the edge of town can see how dire the town’s problems are. Then one day a child goes missing from the Orphan House. At the Mayor’s suggestion, all eyes turn to the Ogress. The Orphans know this can’t be: the Ogress, along with a flock of excellent crows, secretly delivers gifts to the people of Stone-in-the-Glen. But how can the Orphans tell the story of the Ogress’s goodness to people who refuse to listen? And how can they make their deluded neighbors see the real villain in their midst?

The Orphan Master's Son

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Author :
Publisher : Random House Incorporated
ISBN 13 : 0812992792
Total Pages : 465 pages
Book Rating : 4.8/5 (129 download)

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Book Synopsis The Orphan Master's Son by : Adam Johnson

Download or read book The Orphan Master's Son written by Adam Johnson and published by Random House Incorporated. This book was released on 2012 with total page 465 pages. Available in PDF, EPUB and Kindle. Book excerpt: The son of a singer mother whose career forcibly separated her from her family and an influential father who runs an orphan work camp, Pak Jun Do rises to prominence using instinctive talents and eventually becomes a professional kidnapper and romantic rival to Kim Jong Il. By the author of Parasites Like Us.

The Price of an Orphan

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Author :
Publisher :
ISBN 13 :
Total Pages : 204 pages
Book Rating : 4.X/5 (4 download)

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Book Synopsis The Price of an Orphan by : Patricia Carlon

Download or read book The Price of an Orphan written by Patricia Carlon and published by . This book was released on 1999 with total page 204 pages. Available in PDF, EPUB and Kindle. Book excerpt: But then Johnnie and Kay are invited on a camping trip. A special treat to give Johnnie another chance to adjust to life in the outback? Or a cunning trap by a ruthless killer?"--BOOK JACKET.

Orphan Island

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Publisher : HarperCollins
ISBN 13 : 0062443437
Total Pages : 288 pages
Book Rating : 4.0/5 (624 download)

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Book Synopsis Orphan Island by : Laurel Snyder

Download or read book Orphan Island written by Laurel Snyder and published by HarperCollins. This book was released on 2017-05-30 with total page 288 pages. Available in PDF, EPUB and Kindle. Book excerpt: A National Book Award Longlist title! "A wondrous book, wise and wild and deeply true." —Kelly Barnhill, Newbery Medal-winning author of The Girl Who Drank the Moon "This is one of those books that haunts you long after you read it. Thought-provoking and magical." —Rick Riordan, author of the Percy Jackson series In the tradition of modern-day classics like Sara Pennypacker's Pax and Lois Lowry's The Giver comes a deep, compelling, heartbreaking, and completely one-of-a-kind novel about nine children who live on a mysterious island. On the island, everything is perfect. The sun rises in a sky filled with dancing shapes; the wind, water, and trees shelter and protect those who live there; when the nine children go to sleep in their cabins, it is with full stomachs and joy in their hearts. And only one thing ever changes: on that day, each year, when a boat appears from the mist upon the ocean carrying one young child to join them—and taking the eldest one away, never to be seen again. Today’s Changing is no different. The boat arrives, taking away Jinny’s best friend, Deen, replacing him with a new little girl named Ess, and leaving Jinny as the new Elder. Jinny knows her responsibility now—to teach Ess everything she needs to know about the island, to keep things as they’ve always been. But will she be ready for the inevitable day when the boat will come back—and take her away forever from the only home she’s known? "A unique and compelling story about nine children who live with no adults on a mysterious island. Anyone who has ever been scared of leaving their family will love this book" (from the Brightly.com review, which named Orphan Island a best book of 2017).

An Orphan No More

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Publisher :
ISBN 13 : 9780974238340
Total Pages : pages
Book Rating : 4.2/5 (383 download)

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Book Synopsis An Orphan No More by : Rocky Fleming

Download or read book An Orphan No More written by Rocky Fleming and published by . This book was released on 2019-04-15 with total page pages. Available in PDF, EPUB and Kindle. Book excerpt: Even though fully adopted by God the orphan spirit often remains in a Christian. This is the story of a man who struggled with this old identity and overcame it to fully embrace his sonship that God had given to him.

The Red Cross Orphans (The Red Cross Orphans, Book 1)

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Author :
Publisher : HarperCollins UK
ISBN 13 : 0008492379
Total Pages : 341 pages
Book Rating : 4.0/5 (84 download)

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Book Synopsis The Red Cross Orphans (The Red Cross Orphans, Book 1) by : Glynis Peters

Download or read book The Red Cross Orphans (The Red Cross Orphans, Book 1) written by Glynis Peters and published by HarperCollins UK. This book was released on 2021-11-30 with total page 341 pages. Available in PDF, EPUB and Kindle. Book excerpt: From the internationally bestselling author of The Secret Orphan comes her brand new unputdownable historical fiction novel!

The Orphan Mother

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Author :
Publisher : Grand Central Publishing
ISBN 13 : 0446576131
Total Pages : 318 pages
Book Rating : 4.4/5 (465 download)

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Book Synopsis The Orphan Mother by : Robert Hicks

Download or read book The Orphan Mother written by Robert Hicks and published by Grand Central Publishing. This book was released on 2016-09-13 with total page 318 pages. Available in PDF, EPUB and Kindle. Book excerpt: An epic account of one remarkable woman's quest for justice from the New York Times bestselling author of The Widow of the South and A Separate Country. In the years following the Civil War, Mariah Reddick, former slave to Carrie McGavock--the "Widow of the South"--has quietly built a new life for herself as a midwife to the women of Franklin, Tennessee. But when her ambitious, politically minded grown son, Theopolis, is murdered, Mariah--no stranger to loss--finds her world once more breaking apart. How could this happen? Who wanted him dead? Mariah's journey to uncover the truth leads her to unexpected people--including George Tole, a recent arrival to town, fleeing a difficult past of his own--and forces her to confront the truths of her own past. Brimming with the vivid prose and historical research that has won Robert Hicks recognition as a "master storyteller" (San Francisco Chronicle)./DIV