According to a recent article, Castle Creek Inc. has acquired Novavita Thera Inc. in an effort to expand its research and development efforts. Castle Creek Inc. Castle Creek Biosciences Inc.…
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…
This past week, Cycle Pharma announced FDA approval of a once daily dose of NITYR for patients with hereditary tyrosinemia type 1 (HT-1). According to its prescribing information, NITYR…
Tyrosinemia is a metabolic disorder which prevents the body from processing the amino acid tyrosine. Without treatment, the disorder can be lethal. There are three different types of tyrosinemia with…
By Caitlin Seida from In The Cloud Copy CRISPR-Cas9, often called just CRISPR, is a technique used to quickly edit DNA to alter gene functions. Think of it like the…
India first drafted a national rare disease policy back in 2017. Unfortunately, it was never enacted due to budgeting and implementation issues. This draft included suggestions such as a corpus…
September is Tyrosinemia Awareness Month! The good people of the Network Of Tyrosinemia Advocates (N.O.T.A.) have organized a family weekend later this month aimed to bring the Tyrosinemia community together. Let's…
The Advisory Committee on Heritable Disorders is hosting a webinar on September 24, as part of its mission to reduce morbidity and mortality in newborns and children who have, or are…
There is a great unmet medical need for patients living with metabolic disorders. There are few approved treatments for these conditions and many are fatal. These diseases include phenylketonuria, homocystinuria, tyrosinemia, and maple syrup…
According to a story from Business Wire, the drug developer Cycle Pharmaceuticals recently announced that is has completed its goal to obtain the necessary data regarding the stability of its…
Gene editing has come a long way as a treatment option in the last several years. Rapid advances in technology continue to find more and more novel therapies. According to…
According to a story from medicalxpress.com, a team of scientists at the University of Massachusetts Medical School have successfully developed a strategy for editing genes that could be used to…
It's a parent's worst nightmare-- a rare disease that strikes early in life, and without treatment, can cause a patient's death. Tyrosinemia type 1 (TYR1), is a form of a…
According to a story from biomedcentral.com, doctors have begun screening infants for tyrosinemia type 1. However, it is still unclear how accurate the screening process for this genetic disorder is.…
"Mom! What’s for dinner?" It’s a question that strikes fear in the hearts of matriarchs everywhere. Meats, breads, vegetables—you know the basics. Now add in a picky eater or someone…
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…
Tyrosinemia is a hereditary disease that causes a disruption in the body's ability to break down tyrosine, an amino acid. The liver lacks the correct enzyme and, as a result, it…
Mahatma Gandhi taught that we should endeavor to be the change that we wish to see in the world. (I believe Gandhi may have been on to something). I'm wondering…
¿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…
La vida con una grave enfermedad afecta a más de la salud física; salud mental a menudo sufre, también. Que es una de las razones Anna Barlow tomó nota cuando…
Low-fat, low-tyrosine recipes don’t mean you have to go light on the flavor. Because children who have to adhere to a tyrosinemia diet cannot eat foods that include meat, poultry,…
Congrats Patient Worthians! You made it to Friday! This week we have some interesting Myasthenia Gravis pain remedies. Additionally, we have a pretty sweet Acromegaly poster designed by one of our…
Carri Levy’s article about a New Jersey couple John and Amanda Miller, is one of many articles and blogs I’ve read about the Millers; it’s really struck a chord with…
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