Most patients diagnosed with Homocystinuria (HCU), a rare metabolic disorder, don't receive their diagnosis until later in life. Scientists were at a loss for why patients with phenylketonuria (PKU), a similar…
Press releases earlier this year on biotech company, Synlogic, announced quite favorable results on both pre-clinical and clinical data on a unique application of synthetic biology for the rare disease, phenylketonuria (PKU). In just…
According to a story from einnews.com, the cell therapeutics company American Gene Technologies (AGT) has recently announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation…
Today is National Phenylketonuria (PKU) Awareness Day! The United States Senate only very recently voted to designate December 3rd as PKU Awareness Day, in 2012. So let's take a moment…
According to a story from cnbc.com, a group of volunteers in a clinical study drank a concentrated brew that was filled with bacteria. This may sound unpleasant or even dangerous,…
According to a report by the New York Times, a group of researchers tested the health benefits of bacteria this summer. Specifically, researchers aimed to determine if they were able…
According to a story from Shawnee Mission Post, the nonprofit organization RareKC is working to support people with rare diseases that are living the Kansas City area. When living with…
The biotechnology company Synlogic announced positive data from its most recent Phase 1/2a clinical trial of its experimental product SYNB1618. This trial did not test the therapeutic action of the…
Patients living with PKU (phenylketonuria) have hope as researchers look into new drugs that may help the body break down phenylalanine. PKU is an genetic disease in which the body lacks…
The National Society for Phenylketonuria (NSPKU) is holding their PKU Diet Challenge today. The diet challenge is specifically aimed at MPs (Members of Parliament in the UK-- aka, not me),…
On June 28th, 2018, the National Society for Phenylketonuria (NSPKU) is holding a special event that is meant to challenge Members of Parliament (MPs) across the UK. This challenge is…
According to BioPortfolio, there was recently an announcement about preclinical data for SYNB1618, which is a Synthetic Biotic treatment that is being assessed right now in a Phase 1/2a clinical trial in…
According to a story from PR Newswire, the pharmaceutical company BioMarin Pharmaceutical Inc. has recently announced that the U.S. Food and Drug Administration as issued approval for their product pegvaliase-pqpz, which…
A potential treatment for phenylketonuria has progressed to clinical trial stage and the first patient has been dosed, reports Business Wire. This follows successful trials of the drug in non-human…
According to a story from fiercebiotech.com, the gene therapy startup Generation Bio managed to fundraise $100 million in a little over a month. In its prior series A round of…
Andy Trapp is a videographer who runs AB Trapp Productions, a small business documenting weddings and family occasions. He has been living with uncontrolled PKU since the age of 8.…
Andy Trapp is a videographer who runs AB Trapp Productions, a small business documenting weddings and family occasions. He has been living with uncontrolled PKU since the age of 8.…
It is absolutely and unequivocally clear : “The PKU diet must include a medical product, usually consumed as a beverage. There are several brands available that are nutritionally suitable for…
Chocolate, chips, steak. These are a few of our favorite things, but for Dublin brothers Mark and Peter Lavery, they are forbidden It doesn't stop there. Cheese, eggs, beans and…
Her name is Jennifer Payne and if you haven't already read about her on Patient Worthy, she's something of a rare disease revolutionary. Her advocacy work on phenylketonuria (PKU) has…
There's a serious, rising issue brewing over health care coverage for Phenylketonuria (PKU) and it's a steaming pile of outrage. If you haven't heard of this rare disease, here's the…
It seems every disease has its day. According to the fabulous website World CP Day, for cerebral palsy (CP) that day is October 6. More than 17 million people around…
Recently on Recombine, a website dedicated to providing up-to-date blog posts about planning a healthy family, a story popped up from a young woman sharing her experiences about being a…
The 1960s were a time of social rebellion and reform. The Civil Rights Movement, the feminist movement, and Vatican II all rose from a growing sense of dissatisfaction with the…
Happy Pre-Thanksgiving Week Friends! It is almost Thanksgiving which is one of our FAVORITE holidays here at Patient Worthy. Why? Because on this holiday, we are focused on gratitude, family and…
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