pediatrics
| 4 min read
An injection of an antisense oligonucleotide in utero improved outcomes for mice with the neurogenetic disease.
| 6 min read
Via a unique regulatory route in the UK, scientists found that a gene therapy restored vision in young kids with a very rare and severe retinal dystrophy.
| 3 min read
A gene therapy targeting organs beyond the brain could improve quality of life and possibly extend survival in children with the neurodegenerative disease.
| 6 min read
Enzyme replacement therapy has been the mainstay of Pompe disease. New and emerging therapies may provide more options for patients.
| 3 min read
The FDA approved two new medications for Niemann-Pick disease type C that improve symptoms, showing the power of community advocacy.
| 4 min read
In some kids, an RSV infection is mild, while others are hospitalized. Natural killer cells might be the reason why.
| 3 min read
Epilepsy onset and seizure severity are linked to variants in the SCN2A gene, potentially leading to improved diagnostics and treatments.
| 9 min read
This year’s laureates have made groundbreaking discoveries from unlocking the secrets of DNA to fighting enteric diseases and cancer.
| 4 min read
A new porcine model for the rare neurological disorder CLN3 Batten disease more faithfully recreates human symptoms and could accelerate drug development.
| 10+ min read
Left out of clinical trials for years, preterm infants have very few drugs developed just for them. Regulators, industry, and neonatologists plan to fix that.
