Contents

Introduction

Part I
Chapter 1: Hello, Joey 1974
Chapter 2: The Treatment Plan 1974
Chapter 3: Case 44: Babies Hospital, New York City 1935
Chapter 4: A New Disease 1936-1937
Chapter 5: An Equal Opportunity Disease 1938-1942
Chapter 6: Christmas Homecoming 1974-1977
Chapter 7: The Sweat Test 1943-1960
Chapter 8: A Tribe of Desperate Parents 1950-1955
Chapter 9: Lessons from Polio 1955-1960
Chapter 10: The Registry 1960-1966
Chapter 11: The Therapist 1977
Chapter 12: A Disease in Search of Ideas 1964-1980
Chapter 13: The Hitman Cometh 1979-1982
Chapter 14: Salt and Water 1970-1981
Chapter 15: Salty Boy 1981-1983
Chapter 16: Birth of an Advocate 1984
Chapter 17: Out of Many, One 1978-1984

Part 2
Chapter 18: The Gene Hunters 1980-1984
Chapter 19: Lucky Number Seven 1984-1985
Chapter 20: Kate 1986
Chapter 21: To Screen or Not to Screen Newborns 1985
Chapter 22: Michigan 1985
Chapter 23: Joey's Long Goodbye 1986
Chapter 24: Mad Pursuit 1987
Chapter 25: The Gene 1989
Chapter 26: Runny, Like Water 1989-1994
Chapter 27: Venture Philanthropy-A New Way to Fund Drug Development 1977-1999
Chapter 28: The Gene Is the Medicine 1989-1991
Chapter 29: Transforming the Lungs 1992-2003
Chapter 30: First-in-Human Trials 1992-1993
Chapter 31: The Beall Curve 1996

Part 3
Chapter 32: The Joey Fund 1989-1999
Chapter 33: A Network for Developing Therapeutic Drugs 1997-1998
Chapter 34: Aurora 1998-1999
Chapter 35: The Gates Open 1987-2000
Chapter 36: A Tale of Four Families-CF in the New Millennium 1999
Chapter 37: The Takeover 2001
Chapter 38: Getting the Band Together 2001-2003
Chapter 39: Pay to Play 2002-2004
Chapter 40: Molecular Architects of Vertex West 2004-2005
Chapter 41: Rat to Man 2003-2004
Chapter 42: A Christmas Gift 2004-2005
Chapter 43: The Lucky Four 2005-2007
Chapter 44: The Doorman Launches the Era of Genetic Medicine 2007-2008
Chapter 45: Disruption Fall 2008
Chapter 46: Tasting like Average People 2010-2012
Chapter 47: Milestones to a Cure 2004-2015
Chapter 48: Tackling the Common Mutation 2004-2013
Chapter 49: What Mutation Are You? 2013-2014
Chapter 50: The Mother of All Deals 2014
Chapter 51: Very Personal Clinical Trials 2021-2017
Chapter 52: The Triple 2015-2017
Chapter 53: The Home Stretch 2018
Chapter 54: The Leftovers 2015-2020
Chapter 55: A New Generation 2017-2020

Epilogue 2019-2020

Acknowledgments
Timeline
Endnotes
About the Author