NOTA, The Network of Tyrosinemia Advocates, held their 4th Annual NOTA Experience, a family conference in September, despite the extraordinary constraints of time, a pandemic, transportation challenges, and the special…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Researchers have succeeded in editing genes to prevent a metabolic disorder called hereditary tyrosinemia type 1 (HT1) in animals while they are still in the womb. The team, who are…
Hosted by NBS Connect and Southeast Regional Collaboration
If dance is the universal language, then what does NC State’s annual Dance Marathon have to say--especially to type 1 tyrosinemia (TYR)? A lot, it turns out. The numbers seem…
If you lost a child to a rare disease and knew there was a 1 in 4 chance that it could happen again, would you try for more children? That…
Tyrosinemia is an inherited metabolic disorder that inhibits the body’s ability to process or break down essential amino acids found in common foods that are consumed. Tyrosinemia Type 1 attacks…
De acuerdo, Dr.House: Tenemos padres por primera vez que afirman su recién nacido es "irritable, inquieto, vómitos explosivos, y se niega a comer." Aunque también decir que el niño tiene…
¿Quién sabía que el colonialismo podría ser una de las principales causas de las enfermedades raras ?! Al menos, ese es el caso de muchos quebequenses. La tirosinemia, que afecta…
Students at North Carolina State aren’t letting the grass grow beneath their feet, knowing their feet can make a difference in a sick child’s life. At a recent dance marathon…
Imagine your child is stuck at the bottom of a long transplant waiting list when he or she desperately needs a new liver. The thought is terrifying--and for many, terrifyingly…
When lives hang in the balance, should the needs of a few trump the bottom line? That question was all too real for one Australian mother, Shanna Druisi. As reported in…
Who knew that good ol' colonialism could be a leading cause of rare diseases?! At least, that's the case for many Quebecers. Tyrosinemia, which affects about 1 in 100,000 babies…
Okay, Dr. House: We've got first-time parents who claim their newborn is "cranky, fussy, projectile vomiting, and refusing to eat." Although they also say the child has a distended stomach,…
If I gave you 10 bucks right now, what would you do with it? Buy a ticket to a movie matinee, maybe, or pick up that new paperback you've been dying…
The Schriever Air Force Base tells the story of Caden Rose, an 18 month year-old diagnosed with a very rare genetic disorder called tyrosinemia type 1. The disorder makes the body unable…
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