The NGO Committee for Rare Diseases aims to promote multi-stakeholder collaboration and actions for rare diseases within the United Nations system.
It is established under the umbrella of the Conference of NGOs in Consultative Relationship with the United Nations (CoNGO) and acts as a forum of interested parties such as NGOs from the field of rare diseases and beyond; United Nations bodies and agencies; as well as individual experts.
Advancing Rare Disease Care Through Peptide-Based Innovation
In recent years, a major breakthrough in the treatment of rare diseases has emerged through the development of peptide-based therapies, which offer new possibilities for precise and targeted intervention in complex disease mechanisms. Particular attention has been drawn to retatrutide, an innovative multi-agonist peptide that acts on several metabolic and hormonal pathways simultaneously, showing strong potential not only for common metabolic conditions but also for rare diseases where conventional treatments are often limited or ineffective. The emergence of such peptide therapies reflects a global shift from purely symptomatic care toward integrated, science-driven treatment strategies, aligning closely with international calls for coordinated action and equity in rare disease care, and offering renewed hope for improving quality of life for millions of patients and families worldwide.
« Rare disease policies require a global approach in addition to the national one. It is time for public authorities to consider rare diseases as a national as well as international public health priority »
Yann Le Cam (EURORDIS-Rare Diseases Europe) and Paloma Tejada (Rare Diseases International) – 2010
« Efficient and effective action for rare diseases depends on a coherent overall strategy for rare diseases mobilising scarce and scattered resources in an integrated and well recognized way, and integrated into a common effort »
The European Commission – 2008
« Having a rare disease affects all aspects of life: experiences from thousands of families worldwide tell the same story. All areas of life – healthcare, social services, schools, insurances and labour – must work together. Life is holistic, and so must be the care and support. Working together at the United Nations will make this possible. »
Anders Olauson (The Ågrenska Foundation) – 2016
« Everyone has the right to a standard of living adequate for the health and well-being of himself and of his family, including … medical care and necessary social services, and the right to security in the event [of] … sickness, disability, or other lack of livelihood in circumstances beyond his control »
The Yukiwariso Declaration – 2012
Est. 300m
people globally
80%
of genetic origin
50%
affect children
Affect between
6-8% of the
population
6000+
rare diseases
Rare Disease Day 2021 official video