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Rare diseases, often referred to as "orphan diseases", affect a small percentage of the population but collectively impact millions of people worldwide. Despite their relatively low prevalence, rare diseases present significant challenges to health systems. These include delays in diagnosis, limited access to specialized care, and insufficient treatment options. Moreover, many of these diseases remain under-researched, and patients often experience social stigma and a lack of adequate support, exacerbating their difficulties.
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In response to these challenges, the World Health Assembly Resolution on rare diseases sponsored by the Arab Republic of Egypt and the Kingdom of Spain and co-sponsored by 25 Member States has placed a global commitment to improving care for persons living with rare diseases. This resolution urges Member States to take comprehensive steps, such as enhancing access to treatments, improving diagnostic capabilities, and investing in research and innovation.
 
It also calls for the development of national strategies and the integration of rare disease considerations into broader health policies to ensure equitable care for all affected individuals. Additionally, the resolution requests the World Health Organization to develop a Global Action Plan on rare diseases, which would serve as a roadmap for coordinated global efforts to address the needs of those living with these conditions.
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Despite the significant progress made following the adoption of the UN Resolution on addressing the challenges faced by those living with rare diseases in 2021, rare diseases continue to remain "invisible" in many national health agendas. Limited recognition at both the policy and funding levels, along with the lack of specialized expertise, continue to create barriers to effective care and innovation.
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