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Andrew Musgrave에서 제공하는 콘텐츠입니다. 에피소드, 그래픽, 팟캐스트 설명을 포함한 모든 팟캐스트 콘텐츠는 Andrew Musgrave 또는 해당 팟캐스트 플랫폼 파트너가 직접 업로드하고 제공합니다. 누군가가 귀하의 허락 없이 귀하의 저작물을 사용하고 있다고 생각되는 경우 여기에 설명된 절차를 따르실 수 있습니다 https://ko.player.fm/legal.
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PERCHERON THERAPEUTICS LTD (PER) - Pioneering Rare Disease Treatment: CEO Dr. James Garner on Transformative Duchenne Muscular Dystrophy Therapies, Strategic Evolution, and FDA Collaborations

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Manage episode 444572185 series 3570035
Andrew Musgrave에서 제공하는 콘텐츠입니다. 에피소드, 그래픽, 팟캐스트 설명을 포함한 모든 팟캐스트 콘텐츠는 Andrew Musgrave 또는 해당 팟캐스트 플랫폼 파트너가 직접 업로드하고 제공합니다. 누군가가 귀하의 허락 없이 귀하의 저작물을 사용하고 있다고 생각되는 경우 여기에 설명된 절차를 따르실 수 있습니다 https://ko.player.fm/legal.

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Explore the transformative journey of Percheron Therapeutics with Dr. James Garner at the helm, as he shares the company's pioneering efforts in the field of rare disease therapies. Discover how their strategic evolution from Antisense Therapeutics to Percheron Therapeutics has positioned them at the forefront of developing groundbreaking treatments for Duchenne muscular dystrophy (DMD). As we delve into the intricacies of their phase 2b trial of avicursen for non-ambulant boys, Dr. Garner reveals the innovative use of the Performance in the Upper Limb (PUL2.0) endpoint and the promising results from their essential non-human primate toxicology study, aligning with FDA requirements for future clinical trials.
Uncover the strategic blueprint Percheron employs to bring their therapies to market, leveraging Ionis Pharmaceuticals expertise and carefully outsourcing critical tasks, allowing them to concentrate on clinical development. Dr. Garner outlines their ambitious commercialization plans, aiming to partner with larger entities for broader market reach. With pivotal data readouts expected soon and ongoing conversations with the FDA, Percheron is on a mission to de-risk their drugs and attract strong partners. Listen in to understand how these innovative therapies could significantly impact the lives of patients with rare diseases, paving the way for a brighter future.

  continue reading

챕터

1. PERCHERON THERAPEUTICS LTD (PER) - Pioneering Rare Disease Treatment: CEO Dr. James Garner on Transformative Duchenne Muscular Dystrophy Therapies, Strategic Evolution, and FDA Collaborations (00:00:00)

2. Developing Therapies for Rare Diseases (00:00:01)

3. Developing and Partnering Drug Therapies (00:12:54)

85 에피소드

Artwork
icon공유
 
Manage episode 444572185 series 3570035
Andrew Musgrave에서 제공하는 콘텐츠입니다. 에피소드, 그래픽, 팟캐스트 설명을 포함한 모든 팟캐스트 콘텐츠는 Andrew Musgrave 또는 해당 팟캐스트 플랫폼 파트너가 직접 업로드하고 제공합니다. 누군가가 귀하의 허락 없이 귀하의 저작물을 사용하고 있다고 생각되는 경우 여기에 설명된 절차를 따르실 수 있습니다 https://ko.player.fm/legal.

Send us a text

Explore the transformative journey of Percheron Therapeutics with Dr. James Garner at the helm, as he shares the company's pioneering efforts in the field of rare disease therapies. Discover how their strategic evolution from Antisense Therapeutics to Percheron Therapeutics has positioned them at the forefront of developing groundbreaking treatments for Duchenne muscular dystrophy (DMD). As we delve into the intricacies of their phase 2b trial of avicursen for non-ambulant boys, Dr. Garner reveals the innovative use of the Performance in the Upper Limb (PUL2.0) endpoint and the promising results from their essential non-human primate toxicology study, aligning with FDA requirements for future clinical trials.
Uncover the strategic blueprint Percheron employs to bring their therapies to market, leveraging Ionis Pharmaceuticals expertise and carefully outsourcing critical tasks, allowing them to concentrate on clinical development. Dr. Garner outlines their ambitious commercialization plans, aiming to partner with larger entities for broader market reach. With pivotal data readouts expected soon and ongoing conversations with the FDA, Percheron is on a mission to de-risk their drugs and attract strong partners. Listen in to understand how these innovative therapies could significantly impact the lives of patients with rare diseases, paving the way for a brighter future.

  continue reading

챕터

1. PERCHERON THERAPEUTICS LTD (PER) - Pioneering Rare Disease Treatment: CEO Dr. James Garner on Transformative Duchenne Muscular Dystrophy Therapies, Strategic Evolution, and FDA Collaborations (00:00:00)

2. Developing Therapies for Rare Diseases (00:00:01)

3. Developing and Partnering Drug Therapies (00:12:54)

85 에피소드

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