Live Breaking News & Updates on Pku

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Synlogic pulls plug after peek at phenylketonuria phase III data

Synlogic Inc. jolted Wall Street with news that the firm is scrapping for futility Synpheny-3, its pivotal study with labafenogene marselecobac (SYNB-1934) for phenylketonuria, and will evaluate strategic options. Shares of the Cambridge, Mass.-based firm (NASDAQ:SYBX) fell 48.7%, or $1.68, to end Feb. 9 at $1.77. Synlogic will cease operations and reduce its workforce by more than 90%, retaining only certain employees to help with the wind-down.

Cambridge , Cambridgeshire , United-kingdom , Nasdaq , Synlogic-inc , Wall-street , Phenylketonuria , Synlogic-inc- , Labafenogene-marselecobac , Synb-1934 , Pku

Death by ZIP Code: How state borders dictate critical screening for newborns

The state where a baby is born determines what serious and rare disorders he or she will be screened for shortly after birth. It’s a patchwork of policies across the country that could have dire consequences. Some parents call it death by ZIP code.

Connecticut , United-states , Aliso-viejo , California , Illinois , Arkansas , Alabama , Hawaii , Capitol-hill , District-of-columbia , South-carolina , North-carolina

Death by ZIP Code: How state borders dictate critical screening for newborns

The state where a baby is born determines what serious and rare disorders he or she will be screened for shortly after birth. It’s a patchwork of policies across the country that could have dire consequences. Some parents call it death by ZIP code.

Connecticut , United-states , Missouri , North-carolina , California , District-of-columbia , Kentucky , Illinois , South-carolina , Oklahoma , Bowling-green , Texas

CNN Newsroom With Alisyn Camerota and Victor Blackwell

alexis beakler says she's terrified because she's running out of a medical grade formula, two of her sons need because of a metabolic condition. alexis is with me now. thank you for spending a few minutes. how much of this formula do you have left, and tell us about pku and why your boys will need to take this formula? >> hi, thanks for having me back. so my two younger sons have pku, which is a metabolic inborn error of metabolism. their bodies can't process one of the amino acids in protein. they can't have anything with protein in it. they get two grams of protein a day, and that's not very much at all. this formula is about 85 to 90% of their daily nutrition because they're so young, so it is critical. we have about i think eleven days left because the company just sent us nine days and thought they did their job, even though we should have had a two-month supply already.

Sons , Alexis-beakler , Two , Condition , Grade-formula , Metabolic , Formula , Boys , Pku , Thanks , Left , Metabolic-inborn-error-of-metabolism

New Day Weekend With Christi Paul and Boris Sanchez

that formula can only be supplied through the manufacturer. she says she's felt helpless because she found out it was on back order and she hasn't gotten any more just yet. those are pictures of three of her boys, two of whom have this problem. alexa is with us now. alexa, thank you so much for taking time to talk to us. listen, anybody out there who has had to be responsible for children understands the fear that would be coming to you day by day right now. help us understand the necessity of this formula for your children. what would happen to them if they didn't have access to it? >> yeah. so this formula is medical formula. it's medically necessary. they'll be on it the rest of their lives everyday. they have a condition called pku. they can't process protein. so, you know, everything has protein in it. so this formula has all of the other amino acids besidies the

Formula , Manufacturer , Two , Order , Boys , Pictures , Three , Us , Children , Anybody , Alexa-smell , Problem

CBS Evening News With Norah ODonnell

those with special dietary needs are growing desperate. 8-year-old izzy bradford is a bundle from injure but suffers from pku. she relies on special formula for about 70% of her nutrition. >> the can you shake it? mm-hmm. >> reporter: what would happen to izzy's body if she didn't have this metabolic formula? >> she would start to break down psychologically, emotionally and physically. it is 70 portion of her nutrition. >> reporter: could it damage her brain. >> it would, long term not having the formula would result in permanent brain damage. >> reporter: about 17,000 americans suffer from pku, one of many metabolic disorders requiring specialized formula for life. how many cans arrived in your last order. >> two cans. >> reporter: how many were supposed to arrive? >> 16. >> reporter: pediatrician dr. paul levy fears if more patrons run dry, treatment will be limited. >> it's going to be a big problem. even if they get sick and we admit them to the hospital, the tools we use are based on the

Formula , Nutrition , Bundle , Needs , Izzy-bradford , Pku , 70 , 8 , Reporter , Body , Metabolic-formula , Brain

Nashville's Best Fries & Baked Potatoes, From A Guy Who Can Only Eat Taters | 1075 The River

A friend of our show has a condition called PKU (greater explanation) he's lived with all his life. There's just a few foods he can eat. Besides some medical formulas, his diet is limited to potatoes. He's eaten french fries and baked potatoes as his only food .

Cheesecake-factory , Waffle-house , Pku , Ries , Otatoes , Ashed-potatoes , Riend , Oods , How , Iet , Cdonalds

Teenager pleads with Matt Hancock to make life-changing drugs available to adults


Now drama student Lily Wood, 19, has penned an emotional letter to Health Secretary Matt Hancock urging him to intervene.
She wrote: "Are you aware of any drug other than Kuvan that is taken away from the patient when they reach 18? I certainly can't.
"All of my life I have been on a highly restrictive low protein diet where I cannot eat meat, fish, eggs, dairy, pulses, and even some fruit and vegetables are restricted.
"If PKU sufferers don't follow this diet the side effects are immense. Depression, confusion, memory loss, anxiety, motor skills and hospitalisation.
"Patients with type 2 diabetes could be treated with a Mediterranean diet, yet NICE doesn't take their insulin away.

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