Happy Friday, Patient Worthians! This week, we have a video from a PKU patient telling a first-hand account of the deterioration and serious consequences he faced after he went astray…
According to a story from biospace.com, a treatment developed by the pharmaceutical company Pfizer was given Breakthrough Therapy designation by the Food and Drug Administration (FDA). The drug is a…
According to an article from news-medical.net, researchers are beginning to delve into the causes of a rare lung disease called primary ciliary dyskinesia. This research is revealing previously unknown information…
The biotech company Myonexus Therapeutics may soon begin clinical trials for muscular dystrophy gene therapy treatment after obtaining nearly $2.5 million of investments. The research behind the new treatment was…
Just this past week 17-year-old Tina McElligott from Kilflynn, Co.Kerry, passed away from rare disease complications, reported Irish Mirror. She had suffered her entire life with Alpers syndrome, having consistent and intense…
Nothing prepares families for the myriad of tests with strange sounding names that are required when they start to look for solutions to the problems brought about by a member's…
Elizabeth Jane Allen, known best as "Betty", will be honored this week for the wonderful woman she was and the lasting impact she has made on the cystic fibrosis community, reports…
Spark Therapeutics will be hosting a conference call this Tuesday. For those interested in the business side of therapy, this is an interesting opportunity to observe. Find out more about…
According to an announcement from ema.europa.eu, the European Medicines Agency (EMA) recently granted market authorization for a new treatment for alpha-mannosidosis. The new drug is an enzyme replacement therapy called…
Maddie Holt from Everett Washington is a 5-year-old with severe disabilities. She can barely hear or see, and she certainly can't walk or talk, reports Maine Public. Unfortunately for her,…
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.…
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