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Positive and negative controls for ssASOs and Gapmers that can be used for in vitro tox studies

N1C Preclinical Workgroup: Preclinical Modeling Systems

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N1C Preclinical Workgroup: Ordering ASO Checklist

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N1C Preclinical Workgroup: Valuable Tools for the Preclinical Stage of Developing Potential Therapeutics

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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...

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N1C Patient Identification Workgroup: Compilation of Useful Tools

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

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‘This is not a science problem anymore': Paths emerge for scaling up rare disease medicine

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MAKING GENETIC THERAPIES AFFORDABLE AND ACCESSIBLE

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Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N=1 Exon Skipping ASOs

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

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Nonclinical Testing Draft Guidance

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

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Administrative and Procedural Draft Guidance

IND Submissions for Individualized Antisense Oligonucleotide Drug Products: Administrative and Procedural Recommendations Guidance for...

Positive and negative controls for ssASOs and Gapmers that can be used for in vitro tox studies

N1C Preclinical Workgroup: Preclinical Modeling Systems

N1C Preclinical Workgroup: Ordering ASO Checklist

N1C Preclinical Workgroup: Valuable Tools for the Preclinical Stage of Developing Potential Therapeutics

Community Job Openings

N1C Patient Identification Workgroup: Compilation of Useful Tools

Community Events

‘This is not a science problem anymore': Paths emerge for scaling up rare disease medicine

The Cost of Delayed Diagnosis in Rare Disease: A Health Economic Study

All of Us and the Promise of Precision Medicine:

MAKING GENETIC THERAPIES AFFORDABLE AND ACCESSIBLE

Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N=1 Exon Skipping ASOs

Illustrated Example for n=1 Clinical Protocol

Illustrated Example for n=1 Consent Form

Clinical Recommendations Draft Guidance

CMC Recommendations Draft Guidance

Nonclinical Testing Draft Guidance

Administrative and Procedural Draft Guidance

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