A Delaware County infant with a rare metabolic disease, CPS1 deficiency, was treated at CHOP with a custom CRISPR therapy.

Prader-Willi syndrome is a rare genetic disease that causes poor feeding in infancy but later triggers insatiable hunger.

An infant with a rare urea cycle disorder became the first patient to receive a personalized gene-editing therapy. His care ...

KJ received the bespoke CRISPR treatment 6.5 months after birth, receiving three infusions of the therapy as of April 2025.

Dopamine deficiency occurs when levels of the neurotransmitter dopamine are low, affecting movement, emotions, sleep, and ...

The goal is to reuse key parts of the treatment and simply swap in a custom set of instructions for each patient's specific ...

Deficiency can lead to various health issues like high blood pressure, muscle twitches, fatigue, and even mental health conditions. Addressing magnesium levels through diet or supplementation is ...

Contact Alex at [email protected] AI-generated accounts impersonating people with Down syndrome are spreading across social media, according to an analysis by CBS News Confirmed. Many of ...

When accompanied by symptoms like irritability, memory problems, depression, and dementia-like changes, these specific cravings strongly suggest B12 deficiency. "Because these effects are broad ...

Every day, doctors across various specialties diagnose cases of inflammatory arthritis, enteropathy, hyperinsulinemic ...

Having a larger waistline, high blood pressure and other risk factors that make up metabolic syndrome is associated with an increased risk of young-onset dementia, according to a new study.