
Saving Ryan
Emil D Kakkis
Saving Ryan is the inspiring story by physician-scientist Dr. Emil Kakkis about his journey to develop a new, first-ever treatment for an ultra-rare genetic disease called MPS. The scientist had great challenges getting grant and industry support for this rare disease despite the solid science behind doing the treatment. Simultaneously across the country, Mark Dant was in a quest to save his 9-year-old son, Ryan, afflicted by MPS for which there was no cure. Through extraordinary persistence, and with a clock-ticking on Ryan’s declining health, Mr. Dant eventually learns of Dr. Kakkis and his work, whose enzyme replacement therapy could potentially treat Ryan successfully. With renewed hope of saving his son’s life, Mr. Dant turns his attention to fundraising through his own Ryan Foundation to support the continued development of the life-saving treatment. Through an extraordinary series of obstacles and heartaches, Dr. Kakkis develops a successful treatment and ultimately gains the required support of a biotech company to complete the project. Despite early success treating MPS patients, the FDA’s abruptly changing policies caused great uncertainty as to whether the therapy would ever get approved – leading to a dramatic showdown at an FDA Advisory Committee meeting.
Duration - 13h 7m.
Author - Emil D Kakkis.
Narrator - Michael Curtis.
Published Date - Sunday, 22 January 2023.
Location:
United States
Description:
Saving Ryan is the inspiring story by physician-scientist Dr. Emil Kakkis about his journey to develop a new, first-ever treatment for an ultra-rare genetic disease called MPS. The scientist had great challenges getting grant and industry support for this rare disease despite the solid science behind doing the treatment. Simultaneously across the country, Mark Dant was in a quest to save his 9-year-old son, Ryan, afflicted by MPS for which there was no cure. Through extraordinary persistence, and with a clock-ticking on Ryan’s declining health, Mr. Dant eventually learns of Dr. Kakkis and his work, whose enzyme replacement therapy could potentially treat Ryan successfully. With renewed hope of saving his son’s life, Mr. Dant turns his attention to fundraising through his own Ryan Foundation to support the continued development of the life-saving treatment. Through an extraordinary series of obstacles and heartaches, Dr. Kakkis develops a successful treatment and ultimately gains the required support of a biotech company to complete the project. Despite early success treating MPS patients, the FDA’s abruptly changing policies caused great uncertainty as to whether the therapy would ever get approved – leading to a dramatic showdown at an FDA Advisory Committee meeting. Duration - 13h 7m. Author - Emil D Kakkis. Narrator - Michael Curtis. Published Date - Sunday, 22 January 2023.
Language:
English
Opening Credits
Duration:00:00:10
Chapter 00: Foreward
Duration:00:05:05
Chapter 01: The Moment Of Truth
Duration:00:36:42
Chapter 02: A Bake Sale For Ryan
Duration:00:38:15
Chapter 03: Liz and the Mistaken Mixing Experiment
Duration:00:22:59
Chapter 04: Never Treated Before
Duration:00:25:51
Chapter 05: Bungalow Beginnings
Duration:00:26:49
Chapter 06: Shoestring Science
Duration:00:21:29
Chapter 07: The Search For Money
Duration:00:25:54
Chapter 08: The Power Of Friends
Duration:00:26:00
Chapter 09: The Dants Find Their Doctor
Duration:00:10:34
Chapter 10: A Key Connection
Duration:00:26:32
Chapter 11: Building A Team That Dreams
Duration:00:30:37
Chapter 12: A Better Batch
Duration:00:33:04
Chapter 13: Meet The FDA
Duration:00:13:15
Chapter 14: Pressures and Protocols
Duration:00:17:26
Chapter 15: Finding The Right Patients
Duration:00:18:51
Chapter 16: The Impossible Finally Happens
Duration:00:16:55
Chapter 17: "Hell Week" and Happiness
Duration:00:29:30
Chapter 18: Crisis During An Infusion
Duration:00:20:06
Chapter 19: Finding My Purpose
Duration:00:28:22
Chapter 20: The New FDA: A Bubble Bursts
Duration:00:35:16
Chapter 21: Double Blind, Double Trouble
Duration:00:10:40
Chapter 22: Hold For The Senator
Duration:00:08:26
Chapter 23: The Precious Nature Of Life
Duration:00:07:21
Chapter 24: Compassionate Abuse
Duration:00:26:46
Chapter 25: The Multi-Million Dollar Gamble
Duration:00:26:37
Chapter 26: Socks Stay On: When Good Is Not Good Enough
Duration:00:22:06
Chapter 27: Decision Day
Duration:00:18:43
Chapter 28: Stating Our Case
Duration:00:28:43
Chapter 29: The Patients Get Their Say
Duration:00:48:10
Chapter 30: The Vote For Life
Duration:00:17:55
Chapter 31: Epilogue
Duration:01:00:21
Acknowlegements by Dr. Emil Kakkis
Duration:00:01:57
Ending Credits
Duration:00:00:21