Argentina has documented over 1,200 cases of inborn metabolic disorders annually, with phenylketonuria being the most ...

The restructuring and other charges were a result of the Company's decision in February 2024 to discontinue Synpheny-3, its pivotal study of labafenogene marselecobac (SYNB1934) in phenylketonuria ...

NGGT Inc., ("Next Generation Gene Therapeutics" or "NGGT"), a clinical-stage biotechnology company pioneering the next wave of gene therapy innovation for genetic rare diseases, today announced ...

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

The U.S. unit of Indian drugmaker Dr. Reddy’s Laboratories is recalling 331,590 bottles of cinacalcet tablets used to treat hyperparathyroidism due to the presence of a known carcinogen. | The U.S.

Group 2 encompasses diseases that need long-term or lifelong management but have relatively lower treatment costs, such as ...

Completed Enrollment of the Phase 3 RISE UP Study of Mitapivat in Sickle Cell Disease; Topline Data from 52-Week Study Expected in Late 2025 –</ ...

Last year, biotech shutdowns spiked. Thankfully, 2024’s numbers are a little lower, dropping from the 27 closures in 2023 to 22 this year.

BioMarin posts Q3 EPS of $0.91, nearly doubling last year's $0.46 and topping estimates. Sales rose 28%, driven by strong ...

Enzymes are substances in the body that cause and speed up crucial chemical reactions. Enzymes’ function is to help trigger bodily processes ranging from digestion to blood clotting to growth. There ...

People were challenged to solve clues hidden on landmarks and buildings around the Tyne Valley village of Corbridge, as part of a treasure hunt to raise awareness of Phenylketonuria (PKU). Chris and ...

WARREN, N.J., Oct. 1, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the FDA has accepted for filing the New Drug Application (NDA) of sepiapterin for the treatment of ...