Robin Fields ProPublica is a nonprofit newsroom that investigates abuses of power. Sign up to receive our biggest stories as ...

Gene therapy represents a paradigm shift in modern medicine, offering unprecedented opportunities to combat diseases at their ...

Dravet syndrome and other developmental epileptic encephalopathies are rare but devastating conditions that cause a host of ...

The five-year trial saw patients who received a single injection of Lumevoq sustaining clinical recovery in a rare condition ...

Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or ...

Organoids were cultured from healthy donor iPSC sources and differentiated using the human iPSC-derived Cerebral Organoid ...

Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or ...

Cell and gene therapies can be life-changing for patients with certain conditions, but the process of receiving them poses barriers for patients and caregivers that require multistakeholder solutions, ...

Older women could be vulnerable to harmful inflammation from new gene therapies to treat incurable eye diseases, new research ...

Researchers developed an epigenetic editing approach to silence the PCSK9 gene, achieving long-term cholesterol reduction ...

Orchard Therapeutics has tasked Robin Kenselaar with accelerating growth of Lenmeldy, the world’s most expensive drug, ...

Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is ...