Resources
Empowers the rare disease patient community to advocate for impactful, science-driven legislation and policy that advances the equitable development of and access to lifesaving diagnoses, treatments, and cures.
Globally connected community committed to eliminating the challenges of rare disease. Global Genes is committed to providing information, resources and connections to all communities affected by rare diseases.
The National Organization for Rare Disorders (NORD) offers a database of approximately 1,300 reports on specific rare diseases written in patient-friendly language on its website.
This book poignantly asks the questions; Why do drugs to treat orphan diseases cost so much? How can we ensure they are affordable? How can their effectiveness be responsibly assessed? And how can access to them be expanded internationally?
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The most complete listing of rare diseases in the U.S. is on the website of the NIH Genetic and Rare Diseases Information Center (GARD).
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The AMP Bespoke Gene Therapy Consortium (BGTC) aims to develop platforms and standards that will speed the development and delivery of customized or ‘bespoke’ gene therapies that could treat the millions of people affected by rare diseases.
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Project Sebastian provides support, action, and awareness for all families affected by all rare diseases.
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This roadmap by Global Genes is designed to put you in the driver’s seat and in control of how to move your research forward. Whether you are an individual, advocate, or organization, this roadmap was created to help you better understand some of the potential routes to treatment and some of the key concepts that are part of these processes.
Podcasts
RARECast is a Global Genes podcast hosted by award-winning journalist Daniel Levine. It focuses on the intersection of rare disease with business, science, and policy.
Once Upon a Gene is a podcast that explores the world of raising children with disabilities and rare genetic disorders.