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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.
For additional information about rare disease and the potential of gene therapy for genetic diseases, please visit:
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
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
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.
Learn more at thearmfoundation.org
Advocacy organization funds treatment research and provides individuals with spinal muscular atrophy (SMA) and their families support. 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, health care providers, researchers, and fundraisers.
Learn more at curesma.org
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
Patient-driven alliance of patient organizations and individuals active in the field of rare diseases in Europe and beyond.
Learn more at eurordis.org
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
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
A network of 19 organizations of patients with rare diseases, present in 16 countries in Latin America.
Learn more at ALIBER.org
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.
Learn more at APARDO.org
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.
Learn more at APEC.org/rarediseases
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.
Learn more at rarevoices.org.au
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.
Learn more at globalgenes.org
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
A network of over 80 member organizations representing rare disease patient groups in over 100 countries worldwide.
Learn more at rarediseasesinternational.org