Newborn screening, conducted within the first 24-48 hours post-birth, identifies hidden genetic, metabolic, and endocrine ...

Despite the “unfortunate” failure, William Blair analysts do not believe that the utreloxastat readout will heavily affect ...

Eton Pharmaceuticals has acquired the US rights to Amglidia, a treatment developed for neonatal diabetes mellitus, from France-based biotechnology company AMMTeK. Amglidia, also known as glibenclamide ...

Biomarin has a near $3bn per annum drug franchise, and is profitable. Elliott Investment Management may be attempting to ...

Senator Christopher “Bong” Go, the chairperson of the Senate Committee on Health and Demography, has expressed optimism over ...

NGGT has initiated Phase I/II clinical trials in the U.S. and China to evaluate the safety and efficacy of NGGT002 for the treatment of Phenylketonuria WALNUT CREEK, Calif., Nov. 11, 2024 ...

NGGT has initiated Phase I/II clinical trials in the U.S. and China to evaluate the safety and efficacy of NGGT002 for the treatment of Phenylketonuria WALNUT CREEK, Calif., Nov. 11, 2024 /PRNewswire/ ...

You might be more familiar with the term 'heel prick test' as it was formerly known, but the 'newborn blood spot test' is ...

Reddy’s last issued a voluntary recall back in April when it pulled six lots of sapropterin dihydrochloride powder used to treat phenylketonuria due to potential potency issues following ...

Only two programs are expected to contribute to BioMarin’s top line through label expansions by 2027: Palynziq for adolescent Phenylketonuria (PKU) patients and Voxzogo for Hypochondroplasia (HCH).

Purpose: Blood phenylalanine monitoring is critical for the management of phenylketonuria. We compared three methods for measuring blood phenylalanine concentration: the amino acid analyzer ...

Protein substitutes (PS) are an essential component in the dietary management of phenylketonuria (PKU). PS are available as phenylalanine-free amino-acid mixtures (AAM), glycomacropeptide-based PS ...