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The U.S. Food and Drug Administration ( FDA ) has approved Zolgensma ( Onasemnogene abeparvovec-xioi ), the first gene therapy approved to treat children less than two years of age with spinal muscula ...


Novo Nordisk has announced the submission of a Biologics License Applications ( BLA ) to the US Food and Drug Administration ( FDA ) and a Marketing Authorisation Application ( MAA ) to the European M ...


The results of a pediatric phase 2 clinical trial of Burosumab ( Crysvita ) for the treatment of X-linked hypophosphatemia ( XLH ) in children aged 5 to 12 years were published by the New England Jour ...


Muscle biopsy results from 4053-101 study, a phase 1/2 first-in-human study conducted in Europe to assess the safety, tolerability, pharmacokinetics, and efficacy of Golodirsen in 25 boys with confirm ...


Results from two phase 3 studies of the Tezacaftor ( VX-661 ) / Ivacaftor combination treatment that showed statistically significant improvements in lung function ( percent predicted forced expirator ...


Amyotrophic lateral sclerosis ( ALS ) is a rapidly progressing neurodegenerative disease that is characterized by motor neuron loss and that leads to paralysis and death 2-5 years after disease onset. ...


The FDA ( U.S. Food and Drug Administration ) has approved Spinraza ( Nusinersen ), the first drug approved to treat children and adults with spinal muscular atrophy ( SMA ), a rare and often fatal ge ...


The FDA ( U.S. Food and Drug Administration ) has approved Adynovate [ antihemophilic factor ( recombinant), PEGylated ], an extended circulating half-life recombinant Factor VIII ( rFVIII ) treatment ...


Hypophosphatasia ( HPP ) is a rare inheritable disease that results from loss-of-function mutations in the ALPL gene encoding tissue-nonspecific alkaline phosphatase ( TNSALP ). Therapeutic option ...


Results from the phase II study of WT1 first-in-class immunotherapeutic anti-cancer treatment in multiple myeloma patients following autologous stem cell transplantation ( ASCT ) were presented. Ini ...


Results from a phase 2 clinical trial investigating Palovarotene for the treatment of fibrodysplasia ossificans progressiva ( FOP ) were presented. FOP is an extremely rare, serious disease in which ...


The positive results of the first pivotal phase 3 study of the investigational medicine Epidiolex ( Cannabidiol or CBD ) for the treatment of Dravet syndrome were announced. In this study, Epidiolex ...


The FDA ( Food and Drug Administration ) has approved Vonvendi ( von Willebrand factor [ recombinant ] ). Vonvendi is the first and only recombinant treatment for adults living with von Willebrand Dis ...


The FDA ( Food and Drug Administration ) has granted Breakthrough Therapy designation to Olipudase alfa. This enzyme replacement therapy is being investigated for the treatment of patients with non-ne ...


Using combinations of well-known approved drugs has for the first time been shown to be potentially safe in treating a rare disease. The study has also shown some promising preliminary results for th ...