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Challenges in research and development

Access to effective treatment for rare diseases remains a significant challenge, with less than 5% of the 7,000-8,000 known rare diseases having available therapies. Even when correctly diagnosed, most patients face a lack of approved treatments, with only about 1 in 10 receiving disease-specific care. The few drugs that are available are often prohibitively expensive, further complicating access and placing a heavy burden on resources.

Diagnosis of rare diseases

Early diagnosis of rare diseases is often delayed due to limited awareness among primary care physicians and inadequate diagnostic resources.

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Challenges in research and development

Access to effective treatment for rare diseases remains a significant challenge, with less than 5% of the 7,000-8,000 known

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Prohibitive cost of treatment

The high cost of treating rare diseases, known as "orphan diseases," creates significant barriers to access. Due to small patient populations, drug manu...

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The Indian Scenario

In India, data on the prevalence of rare diseases is scarce, with most cases identified at tertiary hospitals. This lack of epidemiological data hinders understanding

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