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Resources

We have gathered the valuable resources below in one publicly shared central location.

N1C Workshop Recordings

Publications

Articles & Podcasts

Presentations

Regulatory Guidances

Vlog

N1C Workshop Recordings

Patient Selection Workshop
Preclinical Design Workshop
Clinical Outcome Measures Workshop
Institutional Implementation Workshop

N1C Workshop Recordings

Travel Apps

Publications

Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides, Dec 2022
Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis, Jan 2022
Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide, Dec 2021
OTS Rare Disease N-of-1+ Briefing Document, Jan 2020
Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease, Oct 2019
Drug Regulation in the Era of Individualized Therapies, Oct 2019

Publications

Fashion Magazine

Articles & Podcasts

They Created a Drug for Susannah. What About Millions of Other Patients?
Hopes for Antisense Oligonucleotides Remain Strong Despite Severe Adverse Events in Trials, Dec 2022
We Can Cure Disease by Editing a Person’s DNA. Why Aren’t We?
Science - milasen
STAT - milasen
MIT Tech Review - milasen
Nature Medicine - afinersen
STAT - jacifusen
MIT Tech Review - atipeksen
New York Times - valeriasen
STAT - Individualized Medicines
Genomics England Podcast - From Mila to Millions

Articles & Podcasts

Presentations

Leadership Presentation

Presentations

Discovery Forum 2022 | From Mila to millions
RARE-X Exchange
NHGRI Bold Predictions

Court

Regulatory Guidances

Below are links to the US FDA Draft Guidances on Individualized ASOs issued in 2021:

Administrative and Procedural Draft Guidance
Nonclinical Testing Draft Guidance
CMC Recommendations Draft Guidance
Clinical Recommendations Draft Guidance

Regulatory Guidances

Biotech and Pharma.jpeg

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