List of rare disease organisations

Overview

The list of rare disease organisations comprises various groups and associations dedicated to supporting individuals affected by rare diseases. These organisations play a crucial role in advocacy, research, and providing resources for patients and their families. Rare diseases, often referred to as orphan diseases, are conditions that affect a small percentage of the population. Due to their rarity, these diseases often lack sufficient research and treatment options.

Purpose

Rare disease organisations aim to:

• Raise awareness about rare diseases.
• Advocate for policy changes and funding for research.
• Provide support and resources to patients and their families.
• Facilitate connections between patients, healthcare providers, and researchers.

Notable Organisations

National Organisation for Rare Disorders (NORD)

NORD is a U.S.-based non-profit organisation that provides advocacy, education, and patient assistance programs. It serves as a hub for rare disease information and support.

European Organisation for Rare Diseases (EURORDIS)

EURORDIS is a non-governmental alliance of patient organisations and individuals active in the field of rare diseases in Europe. It works to improve the quality of life of all people living with rare diseases in Europe.

Genetic and Rare Diseases Information Center (GARD)

The GARD is a program of the National Center for Advancing Translational Sciences (NCATS) and the National Human Genome Research Institute (NHGRI) that provides information about rare and genetic diseases.

Rare Diseases International (RDI)

RDI is the global alliance of people living with a rare disease of all nationalities across all rare diseases. It advocates for rare diseases as an international public health priority.

Global Genes

Global Genes is a global non-profit advocacy organisation for individuals and families fighting rare and genetic diseases. It provides educational resources, funding for research, and support for patient advocacy.

Activities

Rare disease organisations engage in a variety of activities, including:

• Organising awareness campaigns and events such as Rare Disease Day.
• Funding and supporting research initiatives.
• Offering educational resources and workshops for patients and healthcare professionals.
• Collaborating with pharmaceutical companies to develop treatments.

Challenges

Organisations face several challenges, including:

• Limited funding and resources.
• Difficulty in reaching and supporting a widely dispersed patient population.
• Navigating complex regulatory environments for drug development.

Conclusion

Rare disease organisations are vital in the fight against rare diseases. They provide essential support to patients and families, advocate for necessary policy changes, and drive research efforts to find cures and treatments.