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Follow the future of this scientific advancement

There are thousands of monogenic diseases (genetic diseases where there is a mutation on just one gene), and each is rare. Because of the promise gene therapy holds in changing how genetic diseases are treated, many groups of scientists across many disciplines are exploring its role in treating monogenic diseases.

North America

Europe

Latin America

Asia Pacific

International

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North America

ASGCT (American Society of Gene & Cell Therapy)

A professional membership organization for scientists, physicians, patient advocates, and other professionals in gene and cell therapy, working in settings such as universities, hospitals, and biotechnology companies. Provides information on the science, technology, and use of gene and cell therapy as well as a Patient Education Program.

Learn more at ASGCT.org

CORD (Canadian Organization for Rare Disorders) 

Works with governments, researchers, clinicians, and industry to promote research, diagnosis, treatment, and services for all rare disorders in Canada.

Learn more at raredisorders.ca

Cure SMA

An advocacy organization that funds treatment research and provides support for individuals with spinal muscular atrophy (SMA) and their families. Their site includes detailed information and resources on the various types of SMA and the current and future genetic research planned for this rare disease for the newly diagnosed, caregivers, healthcare providers, researchers, and fundraisers.

Learn more at curesma.org

genetic-and-rare-diseases-information-center

GARD (Genetic and Rare Diseases Information Center)

A resource for patients and caregivers offering access to current, reliable, and easy-to-understand information about rare or genetic diseases in English or Spanish.

Learn more at rarediseases.info.nih.gov

NORD (National Organization for Rare Disorders)

NORD is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. Their site includes information about opportunities to participate in clinical trials and other studies so that patients and their physicians may decide whether specific studies are appropriate for them.

Learn more at rarediseases.org

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Europe

EURORDIS

Patient-driven alliance of patient organizations and individuals active in the field of rare diseases in Europe and beyond.

Learn more at eurordis.org

Genetic Alliance UK

Works with organizations and individuals to ensure the needs and preferences of all people affected by genetic, rare, and undiagnosed conditions are recognized, understood, and met.

Learn more at geneticalliance.org.uk

SMA Europe

An umbrella organization that brings together 23 SMA patient and research organizations from 22 different countries across Europe to improve the quality of life of people who live with SMA.

Learn more at sma-Europe.eu

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Latin America