Multi-Omics in Rare Disease Studies: Case Examples
Integrating genomics, transcriptomics, proteomics and metabolomics improves rare disease diagnosis and uncovers mechanisms guiding treatment.
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How Compassionate Use Supports Rare Disease Patients
Explains how FDA expanded access gives investigational drugs to rare disease patients, outcomes, barriers and policy solutions.
Ultimate Guide to Orphan Drug Designation Process
Clear guide to FDA orphan drug designation: eligibility, 90-day review, tax credits, fee waivers, and 7-year market exclusivity.
Top Tools for Genotype-Phenotype Analysis
Comprehensive roundup of 10 tools that link genetic variants and clinical phenotypes to speed rare disease diagnosis and personalize care.
Top Tools for Genotype-Phenotype Analysis
Comprehensive roundup of 10 tools that link genetic variants and clinical phenotypes to speed rare disease diagnosis and personalize care.
Top 6 Therapeutic Approaches for Rare Genetic Conditions
Six modern treatments for rare genetic disorders: small molecules, ASOs, gene therapy, genome editing, combos, and AI-personalized drugs.
8 Questions About Patient-Derived Cell Models Answered
How patient-derived iPSCs, organoids, and PDX models improve drug discovery, personalized therapies, and their limits.
Complete Guide to Personalized Medicine for Rare Diseases
Gene editing, ASOs and patient‑derived iPSCs are reshaping rare disease care, enabling faster, tailored treatments and adaptive regulatory pathways.
Three Treatment Paths — How RareLabs Tests Multiple Approaches at Once
When a family comes to RareLabs, one of the first things we explain is this: we don't put all our eggs in one basket. Rare disease research is inherently uncertain — no one can predict in advance which approach will yield the most promising results for any given condition.
From Your Cells to the Lab — How Personalized Disease Models Work
One of the most powerful tools in modern rare disease research is something that would have sounded like science fiction just twenty years ago: taking a patient's own cells and transforming them into a living, testable model of their disease in the laboratory. This technology — known as induced
What Is a Rare Disease — and Why Does Speed Matter?
A rare disease is any condition that affects fewer than 200,000 people in the United States. But the word "rare" can be deeply misleading. There are over 10,000 known rare diseases worldwide, and collectively they affect an estimated 400 million people globally — roughly 30 million in
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.
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.
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.