Sano blog | Lisa Conroy, MPH

precision medicine patient recruitment rare disease genetic testing protocol design

FDA draft guidance expands single-trial effectiveness pathway

In June 2026, the FDA released a revised draft guidance, Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products, that clarif...

precision medicine public health patient recruitment genetic testing population health

Genomic newborn screening: The operational challenge ahead

A Nature News Feature published on 19 August 2026 synthesized the global wave of genomic newborn screening programs and opened with a concrete result. In the GU...

precision medicine patient recruitment rare disease genetic testing

OptiPrime speeds prime editing but patient identification lags

On August 12, 2026, the David Liu lab at the Broad Institute published OptiPrime, a machine-learning model that predicts how well prime-editing guide RNAs (pegR...

precision medicine genetic epidemiology genetic testing

Inherited genetics linked to CAR-T toxicity and expansion

CAR-T therapies carry meaningful safety risks, and toxicity is known to vary with factors including disease characteristics, patient factors and the therapy its...

precision medicine rare disease protocol design patient engagement

Split verdicts: What HOPE-3 exposes about rare disease evidence

On 29 July 29, 2026, the same clinical trial data produced opposite verdicts. The FDA's Cellular, Tissue, and Gene Therapies Advisory Committee voted 9-3 that a...

screen failure patient recruitment rare disease genetic testing patient engagement

Why precision medicine trials fail on the way from protocol to patient

Precision medicine clinical trials are designed by some of the most capable scientists in drug development. The biomarker hypotheses are well-reasoned, the endp...

patient recruitment protocol design health equity

Decentralized trials expand access and diversity at Mayo Clinic

On July 20, 2026, Mayo Clinic reported peer-reviewed evidence that decentralization changes who takes part in clinical research. In a study published in JAMA Ne...

precision medicine patient recruitment health equity

Why polygenic risk scores miss diverse populations

On 20 July 2026, The New York Times reported that the genetic risk tools entering clinical use do not perform equally well across populations. Polygenic risk sc...

biomarkers neurodegenerative disease podcast recap

Podcast recap: Christopher Walsh on how somatic mosaicism rewrites the story of neurodegeneration

For decades, the dominant framework for understanding neurodegenerative disease has centered on inherited risk genes. A growing body of single-cell genomics res...

precision medicine protocol design population health health equity

The All of Us milestone shows precision medicine’s next infrastructure challenge

The NIH’s All of Us Research Program has become the world’s largest integrated genomic and electronic health record database. Its June 30 data release includes ...

Clinical research blog