Sano blog

precision medicine patient recruitment rare disease protocol design

FDA's rare disease framework could center patient identification

An FDA notice seeking public input is under review at the Office of Management and Budget, according to a Citeline report on October 12, 2026. The notice sugges...

Cardiovascular genetic medicine will need a different patient-finding infrastructure

Genetic medicines are changing who cardiovascular trials need to find. Eligibility is increasingly defined by a genotype or a biomarker level, and the people wh...

thought leadership patient recruitment rare disease genetic testing

FDA approves emcitate for MCT8 deficiency

On September 28, 2026, the FDA approved Emcitate (tiratricol) to treat peripheral thyrotoxicosis in patients with MCT8 deficiency, also known as Allan-Herndon-D...

The Genetics Podcast featuring Jenny Hsieh

rare disease podcast recap

Podcast recap: Jenny Hsieh on why women with Danon disease are being missed

Danon disease is X-linked, and much of the research to date has focused on males. In the latest episode of The Genetics Podcast, Patrick is joined by Jenny Hsie...

The Genetics Podcast featuring Jeffrey Barrett

podcast recap AI chronic disease

Podcast recap: Jeffrey Barrett on the chronic disease subtypes hiding in plain sight

In the latest episode of The Genetics Podcast, Patrick is joined by Dr. Jeffrey Barrett, Professor of Computational Predictive Medicine at the University of Hel...

precision medicine patient recruitment rare disease genetic testing

AATD gene editing race hinges on patient identification

In September 2026, three in vivo gene editing programs reported progress against the same rare disease, aimed at the same mutation. On September 7, YolTech repo...

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Parkinson’s patients first to benefit from Sano Genetics and PKB’s new partnership

Sano Genetics and Patients Know Best (PKB) are excited to announce a new partnership to streamline and accelerate clinical trial recruitment processes, launchin...

The Genetics Podcast featuring Inigo Martincorena

podcast recap

Podcast recap: Inigo Martincorena on somatic evolution in healthy tissue

Every cell in the body picks up changes to its DNA over a lifetime. For most of the history of cancer genomics, researchers could only read those changes once a...

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Genetic eligibility shows up earlier in the pipeline than most plans assume

During enrollment planning, genetic eligibility is often treated as a therapy area question, settled once a program knows which organ system it targets. Genetic...

patient recruitment rare disease genetic testing neurodegenerative disease

FDA approves first therapy for Sanfilippo Type A

On 17 September 2026, the FDA granted standard full approval to FAYUVI (rebisufligene etisparvovec-hopf), a single-dose intravenous AAV9 gene therapy from Ultra...

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