Connecting, Collaborating, Communicating, Challenges: Sharing experiences navigating family conversations in an n-of-1 investigational treatment setting
Selecting the right candidate: Which disease-causing variants are eligible for N=1 ASOs?
Tailored RNA therapies for ultrarare CNS diseases & mutations: a criteria framework for patient ID
Fast Track to Final Drug Product: Understanding Costs, Challenges, and Timelines in Personalized Medicine Manufacturing
"Control of starting material quality for oligonucleotide therapeutics" and "To Synthesis and Beyond…Production of oligos for ‘N-of-1’ therapies"
CMC Recommendations Draft Guidance
Preclinical Development for Personalized Therapy Using Disease-Relevant Cell Models
The Most Important Aspects of the Guidelines for N=1 Exon Skipping Design and in vitro Development
Models for Sustainable Discovery
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
A Guide to Chemical Considerations for the Pre-Clinical Development of Oligonucleotides
Strategies to improve the design of gapmer antisense oligonucleotide on allele-specific silencing
The Most Important Aspects of the Guidelines for N=1 Exon Skipping Design and in vitro Development
Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N=1 Exon Skipping ASOs
Nonclinical Testing Draft Guidance
OTS Rare Disease N-of-1+ Briefing Document, Jan 2020
Regulatory Pathway: Where We Are Today
Approach to Drafting Regulatory Submissions and Challenges of Institutional Implementation
Illustrated Example for n=1 Clinical Protocol
Illustrated Example for n=1 Consent Form
Administrative and Procedural Draft Guidance
Clinical Recommendations Draft Guidance
Institutional Implementation Workshop
Treating the Nano-Rare: Redefining Clinical Management, One Patient at a Time
Rapid development of an n-of-few gene therapy for SPG50 through duantless collaboration
Stopping the clock on KIF1A associated neurological disorder
PRAX-222 Therapy for a Child: Prenatal Onset SCN2A Developmental and Epileptic Encephalopathy in a Named Patient Setting
Potential Treatment: CMT2S Caused By IGHMBP2 Cryptic Splice Site Variant With ASO Based Therapeutic
ASO treatment for SCN2A-encephalopathy, allele-specific vs non-specific
Perspectives on Clinical Outcomes for N=1 Patients: Insights from N1C Clinical Outcomes Workgroup
N of Small Trial with LBSL
An Overview of the N=1 Collaborative and ASO Treatment for KCNT1
Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide
A framework for N-of-1 trials of individualized gene-targeted therapies for genetic diseases
N-of-1 Studies in an Era of Precision Medicine
A Multistakeholder Perspective on Advancing Individualized Therapeutics
Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease
Year End Review and Outcome Measures Workshop
Data Sharing & an Open Letter
Treating the Nano-Rare: Redefining Clinical Management, One Patient at a Time
The challenges and opportunities in rare disease
Transforming Patient Access to High-Cost, One-Time Treatments through Financial Engineering Innovation
Stopping the clock on KIF1A associated neurological disorder
Regulatory: The Future Pathway to Access
Regulatory Pathway: Where We Are Today
From Mutation to Dose-able CRISPR Medicine In 3 Months: Obstacles & Actionable Paths Forward
New Business Models and Funding Strategies for Developing and Paying for Rare Diseases
Models for Sustainable Discovery
Joining forces to develop individualized antisense oligonucleotides for patients with brain or eye diseases: the example of the Dutch Center for RNA Therapeutics
Employers Don't Want to Pay for Life-Saving Cures for Rare Diseases
How to pay for individualized genetic medicines
A Multistakeholder Perspective on Advancing Individualized Therapeutics
‘This is not a science problem anymore': Paths emerge for scaling up rare disease medicine
All of Us and the Promise of Precision Medicine:
MAKING GENETIC THERAPIES AFFORDABLE AND ACCESSIBLE
Drug Regulation in the Era of Individualized Therapies
Clinical Recommendations Draft Guidance
CMC Recommendations Draft Guidance
Nonclinical Testing Draft Guidance
Administrative and Procedural Draft Guidance
Discovery Forum 2022 | Julia Vitarello & Timothy Yu | From Mila to millions
Discovery Forum 2022
RARE-X Exchange - The Future is Now: Realizing the Possibilities of Patient Driven Research
Bold Predictions for Human Genomics by 2030: Session 10
