BioMarin (BMRN) announced positive new data from studies of Voxzogo in children with achondroplasia and in ongoing clinical trials ...

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

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 ...

A new study suggests early detection improves outcomes, but metabolic episodes in the first days of life still prove ...

Pluvia AS has synthesized pharmacological chaperones able to stabilize phenylalanine hydroxylase (PAH) reported to be useful for the treatment of hyperphenylalaninemia, particularly phenylketonuria ...

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

Synlogic (NASDAQ:SYBX – Get Free Report) is expected to issue its quarterly earnings data on Tuesday, March 18th. Analysts ...

A nutritionist has warned of the dangers of consuming too many sweeteners instead of sugar. Everyday household sugar, often ...

Nutritionist Maria Merino shared her opinion on the sugar vs sweetner debate in a TikTok video, which was originally posted ...

A nutritionist has warned people to avoid constantly using sweeteners over sugar as she claims the former is 'worse' and can ...

As Rare Disease Day is commemorated on 28 February, experts discuss the barriers to gene therapy access for some rare ...

The first article deals with the problem of using the response to injected phenylalanine to determine whether or not the subject is heterozygous for phenylketonuria. The second article suggests a ...