Q3 2024 Earnings Call Transcript November 7, 2024 PTC Therapeutics, Inc. beats earnings expectations. Reported EPS is $-1.39, ...

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

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/ ...

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

GENEVA, SWITZARLAND / ACCESSWIRE / October 29, 2024 /RELIEF THERAPEUTICS Holding SA (SIX:RLF)(OTCQB:RLFTF)(OTCQB:RLFTY) (Relief, or the Company), a biopharmaceutical company committed to delivering ...

The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...

were associated with the use of sapropterin in children <4 years with confirmed BH4-responsive phenylketonuria or mild hyperphenylalaninemia (Muntau et al., 2017). We found that vomiting may be a ...

Saline drops are safe to use in babies with nasal congestion. They help clear mucus and draw moisture from swollen nasal tissues so your infant or toddler can breathe more comfortably. First, make ...

Oct. 24, 2024 — Early results from 4,000 babies show that genome sequencing picks up many more serious health conditions than standard newborn screening and is ... Birth: It's a Tight Squeeze ...

Scientists reprogrammed a woman's fat cells to become insulin-making beta cells, reversing her type 1 diabetes.

Newborn screening is a fundamental right for every child, crucial for early diagnosis and intervention of preventable ...