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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


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.

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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.

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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.

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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.

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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.

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

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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.

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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.

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Latin America


Latin American Alliance for Rare Diseases

A network of 19 organizations of patients with rare diseases, present in 16 countries in Latin America.

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Asia Pacific


Asia Pacific Alliance of Rare Disease Organisations

Patient advocate leaders from across the Asia Pacific region representing rare diseases and working together on common goals, facilitating research in the region, sharing resources and best practices, and collaborating on joint initiatives.

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APEC Rare Disease Network

With an estimated 200 million people in the APEC region living with one of 7,000 known rare diseases, this project is working to address barriers to diagnosing or managing healthcare services for populations with rare diseases.

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Rare Voices Australia

RVA provides collaborative leadership for the development and implementation of a National Rare Disease Policy in Australia to drive the best outcomes for Australians living with a rare disease.

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ARM Foundation for Cell & Gene Medicine

Created to educate, engage, and empower patients, caregivers, industry leaders, and other stakeholders to help advance the science and benefits of gene therapy, gene editing, cell therapy, tissue-engineering, and organ regeneration.

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Global Genes

Helping rare disease patients find and build communities, gain access to information and resources, connect to researchers, clinicians, industry, government, and other stakeholders, share data and experiences, stand up, stand out, and become effective advocates on their own behalf.

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Rare Diseases International

A network of over 80 member organizations representing rare disease patient groups in over 100 countries worldwide.

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