pharmaphorum6 年
FDA approves new treatment for rare genetic disease PKU
The US Food and Drug Administration (FDA) has approved biotech BioMarin’s Palynziq (pegvaliase-pqpz) for adults with the rare and serious genetic disease phenylketonuria (PKU). Patients with PKU ...
Nature3 年
Better than natural: next-generation therapeutics through advanced protein engineering
PKU is an inborn ... for more widespread diet-related indications, as well as other GI-stable proteins to locally address disorders such as inflammatory bowel disease. Since the first approval ...
pharmaphorum6 年
FDA approves new treatment for rare genetic disease PKU
The US Food and Drug Administration (FDA) has approved biotech BioMarin’s Palynziq (pegvaliase-pqpz) for adults with the rare and serious genetic disease phenylketonuria (PKU). Patients with PKU ...
WebMD6 年
Vitamins by Condition
Considering taking supplements to treat phenylketonuria pku? Below is a list of common natural remedies used to treat or reduce the symptoms of phenylketonuria pku. Follow the links to read common ...
来自MSN23 年
How Tyrosine, an Amino Acid, Works in the Body
It helps generate brain chemicals called neurotransmitters, including dopamine, epinephrine, and norepinephrine. It shows some evidence of improving focus and mental clarity, especially in stressful ...
BBC1 年
Aspartame - is it a possible cause of cancer?
High profile drinks containing aspartame include Diet Coke, Coke Zero ... These are people with an inherited disease called phenylketonuria or PKU. People with PKU are unable to metabolise ...
Business Insider1 个月
Relief Therapeutics Reports Positive Results from RLF-OD032 Proof-of-Concept Clinical Study ...
Phenylketonuria (PKU) is a genetic disorder caused by a deficiency of ... The standard treatment involves a lifelong phenylalanine-restricted diet supplemented with amino acid-based, phenylalanine ...
Study finds one type of food for breakfast 'reduces heart attack and stroke risk'
Going to work on an egg could stave off a heart attack or stroke, according to research. It boosts metabolism - reducing the ...
Relief Therapeutics Reports New Study Results for RLF-OD032 and Files Provisional Patents
Relief Therapeutics Holding SA / Key word (s): Study results Relief Therapeutics Reports New Study Results for RLF-OD032 and Files Provisional Patents 25-Oct-2024 / 07:00 CET/CEST Release of an ad hoc ...
Acsh.org8 天
From a Drop of Blood to a Genome: The Future of Newborn Screening
Today, the GUARDIAN study is moving newborn screening to genomic sequencing, potentially detecting hundreds of conditions before symptoms even appear. But can this cutting-edge technology replace ...
来自MSN9 个月
Metabolic diseases are major health concern. How to prevent them?
Other examples include obesity, Gaucher's disease, phenylketonuria (PKU), medium-chain acyl-CoA dehydrogenase deficiency ... "The cause behind metabolic disease is not always clear as it can stem from ...
PTC Therapeutics Announces FDA Target Regulatory Action Date For Sepiapterin And Lancet ...
"The Lancet publication reflects the promising and transformative nature of the data collected to date on sepiapterin and the therapy's potential to fill the persistent and significant unmet medical ...