Rare Insights: Uncovering The Future Of Rare Disease Treatments

Rare Insights: Uncovering The Future Of Rare Disease Treatments

On “Rare Insights” we bridge the gap between those living with rare diseases and the biopharmaceutical industry. Know Rare amplifies the voices of individuals with rare conditions, providing invaluable perspectives to accelerate therapeutic solutions. Join us as we dive deep into the complexities of rare diseases, exploring real-world insights from passionate industry leaders. Together, we navigate the unknowns and unlock the potential for groundbreaking treatments. Because in this journey, we’re all rare, sharing unique insights and experiences that drive innovation and progress. Our host is Taren Grom, a well-respected media executive, who co-founded and then sold PharmaVoice, the preeminent trade magazine for life science executives at all stages of their careers. PharmaVoice focused on humanizing the people behind the science, a similar sensibility to Know Rare. If you are in the biopharma industry and would like more information about Know Rare, visit knowrare.com/sponsors. Any questions, episode ideas, guest pitches, or comments can be sent into podcast@knowrare.com. For our blog and other podcasts and video content, visit knowrare.com. Be sure to follow us on social media! We are @KnowRare on Instagram, X, and LinkedIn. Please rate and review the podcast on Apple, Spotify, or wherever you listen. This truly helps us climb the charts and allow more bio-pharmaceutical executives and other industry leaders, like yourself, to discover the show. Rare Insights’ team includes Taren Grom, Liz Kay, Kaitlyn Taylor, DNA Today’s Kira Dineen, and Nina Wachsman, CEO and co-founder of Know Rare. We hope you enjoy listening to Rare Insights. Join us to uncover the future of rare disease treatment.

Episodes

September 30, 2024 21 mins

In this episode of "Rare Insights," host Taren Grom speaks with Dr. Joanne Donovah, Chief Medical Officer at Edgewise Therapeutics, about groundbreaking advancements for severe, rare muscle disorders. Dr. Donovan shares why Edgewise’s approach to targeting the basic unit of muscle contraction and being able to preserve and protect muscle function can have a positive impact on both Becker and Duchene patients. This episode also expl...

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In this episode of "Rare Insights," host Taren Grom sits down with Dr. Linda Marbán, Chief Executive Officer at Capricor Therapeutics, to discuss groundbreaking advancements in the treatment of Duchenne muscular dystrophy and other rare diseases through innovative cell and exosome-based therapies. Dr. Marbán shares her journey in the biotechnology field, the unique approach of Capricor’s CAP-1002, and her vision for the future of r...

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In this episode of "Rare Insights," host Taren Grom speaks with Dr. Jay Barth, Chief Medical Officer at Ascidian Therapeutics, about groundbreaking advancements in RNA editing technology and its potential in the rare disease space. Dr. Barth discusses Ascidian’s innovative approach, inspired by “sea squirts,” and shares insights on the development of ACDN-01 for Stargardt disease, an inherited form of macular degeneration. This epi...

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In this episode of "Rare Insights," host Taren Grom sits down with Dr. Kinnari Patel, President, Head of R&D and Chief Operating Officer for Rocket Pharma, to discuss the company’s burgeoning pipeline of rare disease assets and why applying data across its portfolio of potential treatments is just one key to the company’s success.

 

Dr. Kinnari Patel is President, Head of R&D, and Chief Operating Officer for Rocket Pharma, ...

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June 11, 2024 2 mins

“Rare Insights” is a podcast series interviewing industry and organizational leaders about the development of  future therapies for rare diseases.

 

On “Rare Insights” we bridge the gap between those living with rare diseases and the biopharmaceutical industry. Know Rare amplifies the voices of individuals with rare conditions, providing invaluable perspectives to accelerate therapeutic solutions. 

 

Join us as we dive deep into th...

Mark as Played

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