The CFRI CF Community Voices Podcast

The Power of Science, Community and Desperation to Speed Lifesaving Breakthroughs for the Final 10% – Emily Kramer-Golinkoff, MBE

Emily Kramer-Golinkoff, an adult with cystic fibrosis and the Co-Founder of Emily's Entourage, an innovative 501(c)3 foundation that accelerates research for new treatments and a cure for nonsense mutations of CF, describes her experience growing up with CF and the heart-shattering realization that she is a part of the approximately 10% of the CF community that is ineligible for CFTR modulators. She discusses the promise of science and innovation, each individual’s power to create transformative change, and the unique solidarity and commitment of the whole CF community to create a better future for everyone with CF. This presentation was part of CFRI's Virtual 35th National CF Education Conference on July 31, 2022.

CFRI's 35th National CF Education Conference was sponsored by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.

CFRI’s 35th Cystic Fibrosis Education Conference - Awards Celebration on July 30, 2022 (Audio)

At CFRI's virtual Conference Awards Celebration on July 30, 2022, we honored these extraordinary individuals: Richard Moss, MD - Paul M. Quinton Cystic Fibrosis Research Legacy Award; Sonya Haggett, LCSW - CFRI Partners in Living Award In Memory of Anabel Stenzel; Anna Payne – Dave Stuckert Memorial Volunteer of the Year Award; Dennis Nielson, MD, PhD - Professional of the Year Award.

CFRI's 35th National CF Education Conference was sponsored by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.

Keynote Remarks by Janet Woodcock, MD - CFRI’s 35th Cystic Fibrosis Education Conference (Audio)

Dr. Janet Woodcock, Principal Deputy Commissioner of the US Food and Drug Administration, delivered Keynote Remarks at the opening of CFRI’s 35^th National CF Education Conference on July 30, 2022.

CFRI's 35th National CF Education Conference was sponsored by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.

mRNA therapy for People with Cystic Fibrosis: Next Steps – Manu Jain, MD, MSCI (Audio)

Dr. Manu Jain, Professor of Medicine and Pediatrics at Northwestern University’s Feinberg School of Medicine and Director of the Adult CF Program at Northwestern, presents a comprehensive overview on mRNA therapy for people with cystic fibrosis, a mutation-agnostic treatment that could work for any person with CF regardless of their CFTR mutation. Dr. Jain provides an update on the principles behind mRNA therapy, the hurdles it faces to become a viable treatment option, and the various programs in clinical development. This presentation was part of CFRI's Virtual 35th National CF Education Conference on July 30, 2022.

CFRI's 35th National CF Education Conference was sponsored by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.

Advances in Gene Therapy for Cystic Fibrosis – Paul McCray, MD (Audio)

Dr. Paul McCray, Professor of Pediatrics, Division of Pulmonary Medicine, and Professor of Microbiology and Immunology at the University of Iowa, presents an overview on advances in gene therapy for cystic fibrosis, with a focus on gene therapy for lung disease treatment and prevention. Dr. McCray explores the different gene addition and gene editing strategies under investigation and reviews the challenges of delivery to the lung. This presentation was part of CFRI's Virtual 35th National CF Education Conference on July 30, 2022.

CFRI's 35th National CF Education Conference was sponsored by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.

The Power of Science, Community and Desperation to Speed Lifesaving Breakthroughs for the Final 10% – Emily Kramer-Golinkoff, MBE (Audio)

Emily Kramer-Golinkoff, an adult with cystic fibrosis and the Co-Founder of Emily's Entourage, an innovative 501(c)3 foundation that accelerates research for new treatments and a cure for nonsense mutations of CF, describes her experience growing up with CF and the heart-shattering realization that she is a part of the approximately 10% of the CF community that is ineligible for CFTR modulators. She discusses the promise of science and innovation, each individual’s power to create transformative change, and the unique solidarity and commitment of the whole CF community to create a better future for everyone with CF. This presentation was part of CFRI's Virtual 35th National CF Education Conference on July 31, 2022.

CFRI's 35th National CF Education Conference was sponsored by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.

Cystic Fibrosis in Pakistan

In this moving and informative podcast, a personal overview of life with CF in Pakistan is shared by those who live with and treat the disease. Meet two women from Pakistan who share the lack of therapies they have available to treat their disease, and from Dr. Ali Akbar, a pulmonologist in the UK who is working to raise awareness and access to care in his native Pakistan.

If you watch this episode, please let us know what you think at https://www.surveymonkey.com/r/KGB7PKY

CF Community Voices is made possible through grants from Chiesi USA, Genentech, Vertex Pharmaceuticals, Gilead Sciences, and Viatris.

Athletes of the Transplant Games - Part 2

In part 2 of this inspiring podcast, the athletes with CF, all of whom have received double lung transplants, share more about their experiences participating in the Transplant Games. Hear their perspectives on self-care, motivation, and survivor’s guilt. These games provide transplant recipients a chance to honor their donors and demonstrate what their gift means to them. The Transplant games brings out the best in everyone and serves to increase awareness for organ donation. Watch and be inspired!

If you watch this episode, please let us know what you think at https://www.surveymonkey.com/r/KGB7PKY

CF Community Voices is made possible through grants from Chiesi USA, Genentech, Vertex Pharmaceuticals, Gilead Sciences, and Viatris.

Athletes of the Transplant Games - Part 1

In this inspiring podcast, meet several phenomenal athletes with CF, all of whom have received double lung transplants, who share their experiences participating in the Transplant Games. These games provide transplant recipients to honor their donors and demonstrate what their gift means to them. The Transplant games bring out the best in everyone. Watch and be inspired!

If you watch this episode, please let us know what you think at https://www.surveymonkey.com/r/KGB7PKY

CF Community Voices is made possible through grants from Chiesi USA, Genentech, Vertex Pharmaceuticals, Gilead Sciences, and Viatris.

A Conversation on the Impact of Salt in My Soul

In this webinar, recorded on August 23, 2022, Diane Shader Smith, director Will Battersby, and medical ethnographer Rebecca Mueller, PhD, met online for a dynamic discussion about the impact of the documentary film, Salt In My Soul. How has the film impacted viewers globally? What has been its broader impact upon antimicrobial resistance research and awareness? After a roundtable discussion, questions from the audience were answered.

If you watch this episode, please let us know what you think at https://www.surveymonkey.com/r/KGB7PKY

CF Community Voices is made possible through grants from Chiesi USA, Genentech, Vertex Pharmaceuticals, Gilead Sciences, and Viatris.