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Banca dati di FarmacoVigilanza. Banca dati per una ricerca mirata in Medicina. Indice analitico in Medicina. Banca dati sui Farmaci. Banca dati di Medicina. Banca dati di news Headlines. Olipudase alfa, enzyme replacement therapy to treat non-neurological manifestations which characterize Niemann-Pick disease type B. Charcot-Marie-Tooth type 1A disease: PXT3003, a low-dose combination of Baclofen, Naltrexone and Sorbitol, appears safe. Treatment with Eliglustat maintains hematological and organ volume st...

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Rare diseases Xagena News | rarediseasesnews.net Reviews

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Banca dati di FarmacoVigilanza. Banca dati per una ricerca mirata in Medicina. Indice analitico in Medicina. Banca dati sui Farmaci. Banca dati di Medicina. Banca dati di news Headlines. Olipudase alfa, enzyme replacement therapy to treat non-neurological manifestations which characterize Niemann-Pick disease type B. Charcot-Marie-Tooth type 1A disease: PXT3003, a low-dose combination of Baclofen, Naltrexone and Sorbitol, appears safe. Treatment with Eliglustat maintains hematological and organ volume st...

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rarediseasesnews.net rarediseasesnews.net
1

Delta-aminolevulinic acid synthase-1 is a target for additional therapies to control symptoms in acute porphyria | Rare diseases Xagena

http://www.rarediseasesnews.net/articolo/delta-aminolevulinic-acid-synthase-1-is-a-target-for-additional-therapies-to-control-symptoms-in-acute-porphyria

Banca dati di FarmacoVigilanza. Banca dati per una ricerca mirata in Medicina. Indice analitico in Medicina. Banca dati sui Farmaci. Banca dati di Medicina. Banca dati di news Headlines. Delta-aminolevulinic acid synthase-1 is a target for additional therapies to control symptoms in acute porphyria. The basis for the acute symptoms in these diseases has been speculative. In contrast, use of recombinant porphobilinogen deaminase to knock down porphobilinogen in acute porphyria was ineffective. The efficac...

2

Charcot-Marie-Tooth type 1A disease: PXT3003, a low-dose combination of Baclofen, Naltrexone and Sorbitol, appears safe | Rare diseases Xagena

http://www.rarediseasesnews.net/articolo/charcot-marie-tooth-type-1a-disease-pxt3003-a-low-dose-combination-of-baclofen-naltrexone-and-sorbitol-appears-safe

Banca dati di FarmacoVigilanza. Banca dati per una ricerca mirata in Medicina. Indice analitico in Medicina. Banca dati sui Farmaci. Banca dati di Medicina. Banca dati di news Headlines. Charcot-Marie-Tooth type 1A disease: PXT3003, a low-dose combination of Baclofen, Naltrexone and Sorbitol, appears safe. Using combinations of well-known approved drugs has for the first time been shown to be potentially safe in treating a rare disease. The phase 2 randomized clinical trial involved 80 adult patients wit...

3

Prevention and treatment of Pseudomonas aeruginosa bronchial infections in cystic fibrosis patients: Spanish Consensus | Rare diseases Xagena

http://www.rarediseasesnews.net/articolo/prevention-and-treatment-of-pseudomonas-aeruginosa-bronchial-infections-in-cystic-fibrosis-patients-spanish-consensus

Banca dati di FarmacoVigilanza. Banca dati per una ricerca mirata in Medicina. Indice analitico in Medicina. Banca dati sui Farmaci. Banca dati di Medicina. Banca dati di news Headlines. Prevention and treatment of Pseudomonas aeruginosa bronchial infections in cystic fibrosis patients: Spanish Consensus. Neonatal screening and pharmacokinetic / pharmacodynamic knowledge has modified the management of cystic fibrosis-patients. During mild-moderate exacerbations oral Ciprofloxacin ( 2-3 weeks ) can be adm...

4

Alkaptonuria, Nitisinone therapy decreased urinary homogentisic acid excretion to low levels and was well tolerated | Rare diseases Xagena

http://www.rarediseasesnews.net/articolo/alkaptonuria-nitisinone-therapy-decreased-urinary-homogentisic-acid-excretion-to-low-levels-and-was-well-tolerated

Banca dati di FarmacoVigilanza. Banca dati per una ricerca mirata in Medicina. Indice analitico in Medicina. Banca dati sui Farmaci. Banca dati di Medicina. Banca dati di news Headlines. Alkaptonuria, Nitisinone therapy decreased urinary homogentisic acid excretion to low levels and was well tolerated. Alkaptonuria ( AKU ) is a serious genetic disease characterised by premature spondyloarthropathy. Homogentisate-lowering therapy is being investigated for alkaptonuria. For the most efficacious dose, 8 mg ...

5

Children with hereditary tyrosinaemia following newborn screening: Nitisinone improves outcome | Rare diseases Xagena

http://www.rarediseasesnews.net/articolo/children-with-hereditary-tyrosinaemia-following-newborn-screening-nitisinone-improves-outcome

Banca dati di FarmacoVigilanza. Banca dati per una ricerca mirata in Medicina. Indice analitico in Medicina. Banca dati sui Farmaci. Banca dati di Medicina. Banca dati di news Headlines. Children with hereditary tyrosinaemia following newborn screening: Nitisinone improves outcome. The purpose of a study was to describe the outcome of children with HT1 treated with Nitisinone following selective newborn screening ( NBS ) and to compare their outcome with index siblings who had presented clinically. Curre...

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Rare diseases Xagena News

Banca dati di FarmacoVigilanza. Banca dati per una ricerca mirata in Medicina. Indice analitico in Medicina. Banca dati sui Farmaci. Banca dati di Medicina. Banca dati di news Headlines. Olipudase alfa, enzyme replacement therapy to treat non-neurological manifestations which characterize Niemann-Pick disease type B. Charcot-Marie-Tooth type 1A disease: PXT3003, a low-dose combination of Baclofen, Naltrexone and Sorbitol, appears safe. Treatment with Eliglustat maintains hematological and organ volume st...

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