A framework for individualized splice-switching oligonucleotide therapy
Jinkuk Kim, Sijae Woo, Claudio M. de Gusmao, Boxun Zhao, Diana H. Chin, Renata L. DiDonato, Minh A. Nguyen, Tojo Nakayama, Chunguang...
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A young girl’s custom gene therapy hints at a framework for tailored rare disease treatments
Bree Iskandar (STAT) July 12, 2023 When Timothy Yu developed milasen, a custom drug for a young girl named Mila with Batten disease, he...
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What if coding personalized ASO therapies only required the press of a button?
July 10, 2023 12:30-1:30 PM US EDT - ASOs have the potential to become a cornerstone for therapeutic intervention for genetic rare diseases.
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New Business Models and Funding Strategies for Developing and Paying for Rare Diseases
July 17, 2023 12:30-1:30 pm US EST Topics covered will include: How financial portfolio theory can be applied to rare disease therapeutics,
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Perspectives on Clinical Outcomes for N=1 Patients: Insights from N1C Clinical Outcomes Workgroup
June 5, 2023 12:30-1:30 pm US EDT - We will discuss the paradigm shift required to design suitable clinical outcomes for N=1 patients.
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Rare-disease researchers pioneer a unique approach to clinical trials
Mike May (Nature Medicine) May 5, 2023 Randomization and placebo groups are giving way to biomarkers, real-world data from wearables, and...
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Sharing Papers, Presentations and More
April 2023 - Sharing Papers, Presentations and More As the emerging field of individualized medicines grows, the N1C is committed to...
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Tailored RNA therapies for ultrarare CNS diseases & mutations: a criteria framework for patient ID
Tailored n-of-1 RNA treatment approaches targeting ultrarare or even private variants have opened up a qualitatively novel realm of...
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