Meet some of the people featured in the September 2019 issue of The Scientist.

Deep-learning algorithms could help doctors narrow in on the causes of certain medical conditions, say researchers.

Geneticist and profilee Hoda Zoghbi describes her work on rare diseases.

The genetically engineered probiotic, already in clinical trials, may ease patients’ strict dietary regimes.

The medicine increases the activity of a deficient enzyme in certain patients with the condition.

Alnylam’s patisiran interferes with the production of a mutated protein present in people with hereditary transthyretin amyloidosis.

Preliminary data from Sarepta Therapeutics has exceeded expectations, causing a surge in company stock prices this week.

Doctors treated the fetus, who has alpha thalassemia major, with cells from her mother’s bone marrow.

Biotechs have developed enzyme replacements and genetically modified probiotics to treat patients with the rare metabolic disorder.