PTC Therapeutics, Inc. (NASDAQ: PTCT) today shared new data being presented from the Phase 3 APHENITY trial and subsequent open-label extension study at the 2025 American College of Medical Genetics ...

The new data revealed that over 97 percent of trial participants demonstrated the ability to liberalize their diet while on sepiapterin ... for an individual without PKU, while maintaining control ...

PTC Therapeutics (PTCT) shared new data being presented from the Phase 3 APHENITY trial and subsequent open-label extension study at the 2025 ...

Phenylketonuria (PKU ... the body cannot process phenylalanine from the diet effectively. Nerve cells within the brain are exceptionally sensitive to levels of phenylalanine, which explains ...

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.

Medically reviewed by Aviv Joshua, MS, RDN Phenylalanine is an essential amino acid (building block of protein) that the body ...

“L-tyrosine is a precursor to dopamine, the neurotransmitter that aids in motivation, mood regulation, and pleasure,” says ...

Given the chatter that this is a gold-standard ingredient for hair growth, you may be super tempted to hit "add to cart" on ...

PKU is a rare genetic disease that manifests ... At the moment the main treatment strategy is to restrict phe in the diet, a lifetime commitment that often requires nutritional supplements to ...

but we mainly get it from our diet.' Because we all needs biotin ... include biotinidase deficiency (BTD) and phenylketonuria (PKU). The former makes it difficult for the body to reuse biotin.

The diet eliminates artificial colors ... restrictive diets except in rare circumstances involving conditions like phenylketonuria, celiac disease, food allergies, or epilepsy — and then ...