Atlanta-based workers are baffled by Trump administration cuts to research that directly supports departmental health improvement goals.

Synthetic biology specialist Synlogic will start phase 3 testing of a drug for rare metabolic disorder phenylketonuria ... minority of patients and, in the case of Palynziq, can have serious ...

A rare disease in the US is one in 1,500, or fewer than 200,000 people, and an ultra-rare disease is one in 50,000 or fewer than than 6,000 people.

in many cases, multiple factors are necessary for disease development. For example, phenylketonuria requires both a genetic mutation and environmental exposure (dietary phenylalanine) to manifest..

"That has changed," he said, crediting the progress to a national collaborative network connecting hundreds of hospitals that facilitates case transfers, telemedicine, and data sharing. Launched ...

In these cases, whey protein can be a suitable supplement. For example, people with phenylketonuria need to avoid phenylalanine, an amino acid present in many foods. A specific component of whey ...

Gastrointestinal food allergies are not rare in infants and children. Symptoms include vomiting, reflux, abdominal pains, diarrhea and constipation. Clinical diagnosis requires the exclusion of ...

BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PKU ...

Whereas some sites are industry sponsored—BioMarin, in Novato, California, for example, sponsors a site for people affected with phenylketonuria ... In those cases, advertisements have to ...