Rare Disease Treatment Checklist: 7 Things to Consider
Seven-step checklist to choose genetic therapies, use patient-derived models, confirm safety, and plan translation for rare diseases.
John Garcia
No Treatment Options: How Families Find New Solutions
Families facing rare genetic diseases fund research, build registries, use iPSCs and custom therapies when no approved treatment exists.
Small Molecule Drugs vs Gene Replacement: A Comparison
Small molecules give accessible symptom control; gene replacement targets genetic causes but brings higher cost and delivery risks.
What to Ask Before Starting Rare Disease Treatment
Questions to ask before starting rare disease treatment—confirm genetic diagnosis, compare therapies, weigh risks and costs, and plan long-term follow-up.
Gene Therapy vs Antisense Oligonucleotides: Key Differences
Compare gene therapy and antisense oligonucleotides: DNA vs RNA targets, durable vs reversible effects, delivery methods, and best-use cases for rare genetic diseases.
5 Steps to Find Treatment for a Rare Genetic Disease
A 5-step plan to pursue treatment for rare genetic diseases: confirm diagnosis, join advocacy groups, research repurposed drugs, explore personalized therapies, and prepare for trials.
Study Insights: Multi-Modality Drug Discovery
Integrated omics plus AI improve rare-disease diagnosis and target ID, enabling personalized, combination therapies tested in patient-derived models.