blog-icon

Clinical research blog

Explore our blog for insights into the big questions in precision medicine and clinical research.

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...
Continue reading

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...
Continue reading

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...
Continue reading

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...
Continue reading

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...
Continue reading

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...
Continue reading

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...
Continue reading

Podcast recap: Marleah Dean Kruzel on reframing hereditary cancer uncertainty

In the latest episode of The Genetics Podcast, Patrick sits down with Dr. Marleah Dean Kruzel, Professor of Communication at the University of South Florida. He...
Continue reading

Podcast recap: Dave Hallett on cutting through the AI hype in drug discovery

In the most recent episode of The Genetics Podcast, Patrick Short sat down with Dr. Dave Hallett, Chief Scientific Officer at Recursion. Dave has spent nearly t...
Continue reading

Rare disease recruitment problems start earlier than most sponsors think

Rare disease trials have a recruitment problem, but the problem often begins well before recruitment starts.
Continue reading