Your Access to Gene Therapies

Gene therapies can be lifesaving for many patients. But accessing them can be a challenge of its own. Terry and Dr. Bob discuss the private sector’s work to improve accessibility with Biotech executive John Crowley, who shares his views on the moral obligations companies have to patients. Also, Ciji Green shares her battle with her insurance company to access spinal muscular atrophy gene therapy for her daughter.

Guest:

John Crowley, Chairman of the Board, Chief Executive Officer, Amicus Therapeutics

John’s involvement with biotechnology stems from the 1998 diagnosis of two of his children with Pompe disease—a severe and often fatal neuromuscular disorder. In his drive to find a cure for them, he left his position at Bristol-Myers Squibb and became an entrepreneur as the Co-founder, President, and CEO of Novazyme Pharmaceuticals, a biotech start-up conducting research on a new experimental treatment for Pompe disease (which he credits as ultimately saving his children’s lives). In 2001, Novazyme was acquired by Genzyme Corporation where John continued to play a lead role in the development of a drug for Pompe disease as Senior Vice President, Genzyme Therapeutics.

John and his family have been profiled on the front page of The Wall Street Journal and are the subjects of a book by Pulitzer prize-winning journalist Geeta Anand, “The Cure: How a Father Raised $100 Million - And Bucked the Medical Establishment - In a Quest to Save His Children.” The major motion picture, Extraordinary Measures, starring Brendan Fraser and Harrison Ford, is inspired by the Crowley family journey. John also is the author of a personal memoir: Chasing Miracles: The Crowley Family Journey of Strength, Hope, and Joy.

John is a commissioned officer in the U.S. Navy Reserve, assigned to the United States Special Operations Command, and is a veteran of the global war on terrorism, with service in Afghanistan. He graduated with a B.S. in Foreign Service from Georgetown University, and earned a J.D. from the University of Notre Dame Law School, and an M.B.A. from Harvard. 

Hosts:

Terry Wilcox, Executive Director, Patients Rising

Dr. Robert Goldberg, “Dr. Bob”, Co-Founder and Vice President of the Center for Medicine in the Public Interest.

Kate Pecora, Field Correspondent 

Links:

John Crowley

Amicus Therapeutics

‘Maisie’s Army’: How a grassroots group is mobilizing to help toddlers access a lifesaving drug

Patients Rising Concierge 

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The views and opinions expressed herein are those of the guest(s)/ author(s) and do not reflect the official policy or position of Patients Rising.