Sano blog | rare disease (8)

precision medicine rare disease genetic testing

Seeing the future: Breakthroughs in genetic eye disease diagnosis and therapy [Whitepaper]

Genetic eye diseases present significant challenges for patients and healthcare providers alike. Conditions like retinitis pigmentosa, Stargardt disease, and Le...

rare disease genetic testing podcast recap

Podcast recap: Exploring the future of AAV gene therapy with Paul Wille

In the latest episode of the Genetics Podcast, host Patrick Short spoke with Paul Wille, Director of Product Development at Abeona Therapeutics, about adeno-ass...

genetic epidemiology rare disease podcast recap

Podcast recap: How genomics is rewriting the taxonomy of disease with Dr. Lon Cardon

In the latest episode of The Genetics Podcast, host Patrick Short welcomes Dr. Lon Cardon, CEO of the Jackson Laboratory, to discuss his career journey, the ris...

rare disease podcast recap

Podcast recap: Navigating rare disease drug development regulations with Daniel O'Connor

In the latest episode of The Genetics Podcast, Sano CEO Patrick Short sat down with Daniel O’Connor, an expert in regulatory policy for innovative medicines, pa...

patient recruitment rare disease patient engagement

Partnering for progress: The Lupus Nexus Initiative with Lupus Research Alliance

At Sano, we are constantly exploring new ways to use our technology expertise to make a meaningful impact on healthcare. That’s why we’re excited about our part...

rare disease genetic testing population health

Genomic applications in frontline healthcare for at-risk populations

In terms of identifying and supporting at-risk populations, genomics has multiple potential applications in frontline healthcare. While confirmation of an under...

rare disease genetic testing

First-of-its-kind gene therapy restores hearing for toddler with genetic hearing loss

British toddler Opal Sandy has had her hearing restored thanks to a first-of-its-kind gene therapy. Eighteen-month-old Opal received the treatment as part of th...

rare disease genetic testing podcast recap

Podcast recap: Pioneering tRNA therapies with Michelle Werner

In the latest episode of The Genetics Podcast, Sano CEO Patrick Short had a fascinating conversation with Michelle Werner, CEO of Alltrna and CEO/Partner at Fla...

patient recruitment rare disease patient engagement

Webinar recap: Alpha-1 Antitrypsin Deficiency engagement, screening, and retention

In this webinar, "Alpha-1 Antitrypsin Deficiency: Engagement, screening, and retention," Sano Senior Project Manager, Hayley Holt, shares a case study on our wo...

overview of fda guidance on natural history studies - social

rare disease protocol design

Navigating FDA guidance on natural history studies

Developing treatments for rare diseases involves unique obstacles due to the rarity and complexity of these conditions. To address these challenges, the FDA off...

Clinical research blog