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Rare Diseases: Making Treatments Less Rare with Gene Therapy
A resource to explore the discovery of rare diseases and their causative genes | Scientific Data
Who Will Receive Gene Therapy for Rare Diseases?
Gene therapy strategies for rare monogenic disorders with nuclear or mitochondrial gene mutations - ScienceDirect
The Genetic Components of Rare Diseases | The Scientist Magazine®
Overcoming challenges in rare genetic diseases
Ensuring a future for gene therapy for rare diseases | Nature Medicine
Scientists ID Gene Behind Rare Childhood Syndrome | UC San Francisco
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FDA suggests ways to curb constraints with rare disease gene therapy trials - Pharmaceutical Technology
Rare Genetic Diseases
Girl Diagnosed With Rare Genetic Disorder Thanks to Reddit After Years of Inconclusive Testing - ABC News
Rare Disease and Orphan Drugs Journal
Contribution of rare and predicted pathogenic gene variants to childhood-onset lupus: a large, genetic panel analysis of British and French cohorts - The Lancet Rheumatology
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Cancer of the appendix: Very rare, but genes may play role
Ancient gene mutation found to cause rare hereditary condition - NIHR Oxford Biomedical Research Centre
Unique roles of rare variants in the genetics of complex diseases in humans | Journal of Human Genetics
The Wilderness of Rare Genetic Diseases and the Parents Navigating It - The New York Times
Rare Genetic Diseases: Nature's Experiments on Human Development - ScienceDirect
About Rare Genetic Disorders - DDC Clinic
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Rare Genetic Diseases: Nature's Experiments on Human Development - ScienceDirect
