Click below for download: Please feel free to use the ASOs on this list for the toxicity studies in your lab. If you have any ASOs that...

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Welcome to our community job board where we curate relevant job openings to share with our community. If you have a job post to share, we...

Click below for download: Disclaimer: This sample document is the work product of the N=1 Collaborative. This document is intended to...

Updated November 28, 2023 FDA Clinical Investigator Training Course (CITC) 2023: December 6-7 2023 ABOUT THIS EVENT This course is...

In 2014, the National Institutes of Health posted an estimate that at the current pace of drug development and approval, it would take...

This study uses real world data to examine healthcare utilization relative to timing of diagnosis in seven RDs based on anticipated...

Achieving Equitable Access for Federally Qualified Health Center Patients Click below for download: Disclaimer: This document is the work...

The IGI brings together scientists and innovators from diverse disciplines to unlock the potential of CRISPR technology to solve some of...

Antisense oligonucleotides (ASOs) can modulate pre-mRNA splicing. This offers therapeutic opportunities for numerous genetic diseases,...

Click below for download: Disclaimer: This document is the work product of the N=1 Collaborative (the “N1C”), and is offered as an...

Click below for download: Disclaimer: This document is the work product of the N=1 Collaborative (the “N1C”), and is offered as an...

IND Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases:...

Investigational New Drug Application Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating...

Nonclinical Testing of Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases...