Supportive Therapies
There are approved supportive therapies, such as enzyme replacement therapies, that can significantly improve the quality of life and health of patients with rare diseases. These treatments can help manage symptoms and slow disease progression, offering hope to many. However, access to reliable information about these therapies is often limited, making it difficult for patients and their families to find accurate guidance from trusted authorities.
This highlights the urgent need for better awareness, education, and centralized resources to ensure that life-saving treatments are more readily available and understood by those who need them most.
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