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Mashup Score: 1Long-term safety and outcomes in hereditary tyrosinaemia type 1 with nitisinone treatment: a 15-year non-interventional, multicentre study - 3 year(s) ago
Long-term nitisinone treatment was well tolerated and no new safety signals were revealed. Life-limiting hepatic disease appears to have been prevented by early treatment start. Neonatal screening was the most effective way of ensuring early treatment. Standardised monitoring of blood tyrosine, phenylalanine, and nitisinone levels has potential to guide individualised therapy.
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Mashup Score: 1Nitisinone: two decades treating hereditary tyrosinaemia type 1 - 3 year(s) ago
In 1992, Sven Lindstedt and colleagues1 reported the first study using 2-(2-nitro-4-trifluoromethylbenzoyl)-1, 3-cyclohexanedione (nitisinone) to treat five children with hereditary tyrosinaemia type 1. This disorder of tyrosine degradation has a high morbidity and mortality in the early years of life, primarily due to life-threatening hepatic disease including liver failure and hepatocellular…
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Mashup Score: 3Long-term safety and outcomes in hereditary tyrosinaemia type 1 with nitisinone treatment: a 15-year non-interventional, multicentre study - 3 year(s) ago
Long-term nitisinone treatment was well tolerated and no new safety signals were revealed. Life-limiting hepatic disease appears to have been prevented by early treatment start. Neonatal screening was the most effective way of ensuring early treatment. Standardised monitoring of blood tyrosine, phenylalanine, and nitisinone levels has potential to guide individualised therapy.
Categories: Endocrinology, Latest HeadlinesTweet
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Mashup Score: 2Long-term safety and outcomes in hereditary tyrosinaemia type 1 with nitisinone treatment: a 15-year non-interventional, multicentre study - 3 year(s) ago
Long-term nitisinone treatment was well tolerated and no new safety signals were revealed. Life-limiting hepatic disease appears to have been prevented by early treatment start. Neonatal screening was the most effective way of ensuring early treatment. Standardised monitoring of blood tyrosine, phenylalanine, and nitisinone levels has potential to guide individualised therapy.
Categories: Endocrinology, Latest HeadlinesTweet
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Mashup Score: 1Nitisinone: two decades treating hereditary tyrosinaemia type 1 - 3 year(s) ago
In 1992, Sven Lindstedt and colleagues1 reported the first study using 2-(2-nitro-4-trifluoromethylbenzoyl)-1, 3-cyclohexanedione (nitisinone) to treat five children with hereditary tyrosinaemia type 1. This disorder of tyrosine degradation has a high morbidity and mortality in the early years of life, primarily due to life-threatening hepatic disease including liver failure and hepatocellular…
Categories: Endocrinology, Latest HeadlinesTweet
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Mashup Score: 4Long-term safety and outcomes in hereditary tyrosinaemia type 1 with nitisinone treatment: a 15-year non-interventional, multicentre study - 3 year(s) ago
Long-term nitisinone treatment was well tolerated and no new safety signals were revealed. Life-limiting hepatic disease appears to have been prevented by early treatment start. Neonatal screening was the most effective way of ensuring early treatment. Standardised monitoring of blood tyrosine, phenylalanine, and nitisinone levels has potential to guide individualised therapy.
Categories: Endocrinology, Latest HeadlinesTweet
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Mashup Score: 0Nitisinone: two decades treating hereditary tyrosinaemia type 1 - 3 year(s) ago
In 1992, Sven Lindstedt and colleagues1 reported the first study using 2-(2-nitro-4-trifluoromethylbenzoyl)-1, 3-cyclohexanedione (nitisinone) to treat five children with hereditary tyrosinaemia type 1. This disorder of tyrosine degradation has a high morbidity and mortality in the early years of life, primarily due to life-threatening hepatic disease including liver failure and hepatocellular…
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Mashup Score: 1Suitability of nitisinone for alkaptonuria - 3 year(s) ago
In many, but not all, genetic metabolic conditions, biochemical markers are used in the diagnosis and monitoring of patients. However, these markers are not always relevant for the pathogenesis of the respective conditions. For example, in the classical urea cycle disorder citrullinaemia type 1,1 the metabolite citrulline, an amino acid, is greatly elevated in the blood and urine of affected…
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Mashup Score: 1
Nitisinone 10 mg daily was well tolerated and effective in reducing urinary excretion of HGA. Nitisinone decreased ochronosis and improved clinical signs, indicating a slower disease progression.
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Mashup Score: 1
Nitisinone 10 mg daily was well tolerated and effective in reducing urinary excretion of HGA. Nitisinone decreased ochronosis and improved clinical signs, indicating a slower disease progression.
Categories: Endocrinology, Latest HeadlinesTweet
Online—Long-term safety and outcomes in #hereditary #tyrosinaemia type 1 with #nitisinone treatment: a 15-year non-interventional, multicentre study https://t.co/zd7DcYFFqI #RareDisease