How to pay for individualized genetic medicines
Julia M. Y. Pian, Nana Owusu, Julia Vitarello, Winston X. Yan, Andrew W. Lo & Timothy W. Yu (Nature Medicine) June 19, 2024 For precision...
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Building a Bridge Between Rare Disease Patients and the Technologies that Could Help Them
Laura Cowen (Inside Precision Medicine) June 4, 2024 In recent years, whole genome sequencing has made it easier to diagnose rare genetic...
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Preclinical Development for Personalized Therapy Using Disease-Relevant Cell Models
Monday, July 15 2024, 12:30 pm US EDT iXCells Biotechnologies is specialized in providing innovative pre-clinical drug discovery...
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Rapid development of an n-of-few gene therapy for SPG50 through duantless collaboration
June 17, 2024 at 12:30 pm US EDT Steven Gray and Terry Pirovolakis will share the story of the development of a gene therapy treatment...
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Can AI build custom ‘n-of-1’ drugs faster? Maybe not yet, new case study suggests
Jason Mast (STAT News) May 13, 2024 Two years ago, Yiwei She was searching desperately for someone to build a drug for her newborn son,...
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Stopping the clock on KIF1A associated neurological disorder
Rescheduled to September 16, 2024 at 12:30 pm US EDT KIF1A associated neurological disorder is a rare neurodegenerative neurological...
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Fast Track to Final Drug Product: Understanding Costs, Challenges, and Timelines in Personalized Medicine Manufacturing
May 6, 2024 at 12:30 pm US EDT In this webinar, speakers from Berkshire Sterile Manufacturing (BSM) and Argonaut Manufacturing Services...
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Practical Recommendations for the Selection of Patients for Individualized Splice-Switching ASO-Based Treatments
Bianca Zardetto, Marlen C. Lauffer, Willeke van Roon-Mom, and Annemieke Aartsma-Rus on behalf of the Dutch Center for RNA Therapeutics,...
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