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Breaking Barriers: Access to Treatment for Rare Diseases in India

From Treatment to Misdiagnosis: Rare Diseases in the Present Day

Breaking Barriers: Access to Treatment for Rare Diseases in India By Nisha Venugopal, Ph.D. The World Health Organisation (WHO) defines rare diseases as debilitating, lifelong disorders with a prevalence of less than one per 1,000 persons. They include autoimmune disorders, congenital malformations, inherited cancers, and a few endemic infectious diseases that have very low prevalence. …

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FDA Recommendations for Promoting Diversity in Clinical Trial Enrollment

FDA Recommendations for Promoting Diversity in Clinical Trial Enrollment

Over the past few decades, the United States Food and Drug Administration (FDA) has been actively working to promote inclusive enrollment practices aimed at making clinical trials more reflective of the general population; they have also focused on including a larger proportion of demographics most likely to use the medications if approved. Despite broadening eligibility …

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From Treatment to Misdiagnosis: Rare Diseases in the Present Day

From Treatment to Misdiagnosis: Rare Diseases in the Present Day

There are roughly 400 million people affected by rare diseases worldwide, according to the World Health Organization. The historically low awareness and research associated with rare diseases is largely a result of the small amount of people affected worldwide, but rare diseases aren’t “rare” if considered cumulatively. Due to the overall lack of knowledge surrounding …

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Obesity- Regulating Brain Signaling Pathway Genes

Obesity- Regulating Brain Signaling Pathway Genes

The signaling proteins acting in the hypothalamus to direct development of neuronal circuits involved in weight control have been identified by an international research team who have identified rare mutations in the genes for class 3 semaphorins in the severely obese, as published in Cell. Further investigations involving cultured cells and genetically engineered zebrafish and …

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