Sano blog | rare disease (9)

rare disease protocol design

Understanding disease progression: The power of natural history studies

A natural history study is a preplanned observational study designed to track the course of a disease over time. Its purpose is to identify demographic, genetic...

rare disease genetic testing

Exploring natural history studies in drug development

Natural history studies are vital for understanding disease progression, particularly in rare diseases. They provide a baseline to evaluate the effectiveness of...

patient recruitment rare disease

Key takeaways from the World Orphan Drug Congress

Last week, our team had the opportunity to attend the World Orphan Drug Congress in Boston. The event was a dynamic gathering, where leaders from biopharma, reg...

precision medicine rare disease genetic testing

Newborn genome sequencing: Benefits, risks, and ethics

Children born with rare inherited conditions often face a protracted diagnostic odyssey before the cause of their illness is identified. This delay is costly, s...

precision medicine rare disease

Natural history studies: Disease progression and trial design

Natural history studies are instrumental in advancing precision medicine by offering a nuanced understanding of the patient journey across various diseases, inc...

precision medicine rare disease

AI in drug development: What breaks from data to trials

Drug development remains one of the most resource-intensive and failure-prone processes in healthcare. Traditional approaches depend heavily on trial-and-error ...

biomarkers precision medicine rare disease

How natural history studies support rare disease drug development

Natural history studies document disease progression by observing diseases in their unmodified state, deepening our understanding of how conditions evolve over ...

public health rare disease health equity

Newborn screening equity: Sequencing and global gaps

Over the last two decades, following the landmark achievement of sequencing the first complete human genome, a suite of powerful genetic tools has emerged. Gene...

rare disease genetic testing podcast recap

Innovation on Rare Disease Day: Recode CMO John Matthews on changing the future of genetic medicine [podcast]

In observance of Rare Disease Day, our latest episode of the Genetics Podcast presents a fascinating conversation with John Matthews, who was recently promoted ...