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Pseudomonas aeruginosa is the main pathogen in bronchopulmonary infections in cystic fibrosis ( CF ) patients. It can only be eradicated at early infection stages while reduction of its bacterial load ...


Nitisinone ( Orfadin ) has transformed the management of hereditary tyrosinaemia type 1 ( HT1 ). However, the risk of developing hepatocellular carcinoma is related to the age at which treatment is co ...


Alkaptonuria ( AKU ) is a serious genetic disease characterised by premature spondyloarthropathy. Homogentisate-lowering therapy is being investigated for alkaptonuria. Nitisinone ( Orfadin ) decrea ...


Anakinra ( Kineret ) is a recombinant form of interleukin-1 receptor antagonist. It is the drug of choice for Schnitzler syndrome and cryopyrin-associated periodic syndromes. It has also recently be ...


The European Medicines Agency ( EMA ) has recommended Holoclar, the first advanced therapy medicinal product ( ATMP ) containing stem cells, for approval in the European Union ( EU ). Holoclar is a ...


The FDA ( Food and Drug Administration ) has approved Esbriet ( Pirfenidone ) as a treatment for idiopathic pulmonary fibrosis ( IPF ) in the United States. Idiopathic pulmonary fibrosis is a fatal ...


The FDA ( Food and Drug Administration ) has approved Cerdelga ( Eliglustat ) capsules, the only first-line oral therapy for certain adult Gaucher disease type 1 patients. A small number of adult pati ...


Data of the first phase IIIb/IV study, AMBITION, were presented at the ERS International Congress 2014. The study has investigated combination therapy of Ambrisentan and Tadalafil in treatment naïve p ...


Although various modalities of treatment of chronic inflammatory demyelinating polyradiculopathy ( CIDP ) there are not any treatment protocol agreed. Researchers have retrospectively evaluated the ...


The FDA ( Food and Drug Administration ) has announced the approval of Lumizyme ( Alglucosidase alfa ) for treatment of patients with infantile-onset Pompe disease, including patients who are less tha ...


Researchers have compared the phase II and phase III ( EMPOWER ) studies of Dexpramipexole in amyotrophic lateral sclerosis ( ALS ) and have evaluated potential EMPOWER responder subgroups and biomark ...


Tyrosinaemia type 1 ( HT1 ) is a rare disorder of tyrosine metabolism leading to liver failure and hepatocellular carcinoma. Treatment previously consisted of dietary restriction and orthotopic liver ...


A study has assessed the efficacy and safety of Olesoxime, a molecule with neuroprotective properties, in patients with amyotrophic lateral sclerosis ( ALS ) treated with Riluzole ( Rilutek ). A do ...


The neurite outgrowth inhibitor, Nogo-A, has been shown to be overexpressed in skeletal muscle in amyotrophic lateral sclerosis ( ALS ); it is both a potential biomarker and therapeutic target. A ...


The FDA ( Food and Drug Administration ) has approved Cerdelga ( Eliglustat ) capsules, the only first-line oral therapy for certain adult Gaucher disease type 1 patients. A small number of adult pa ...