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amyloidosis

This page shows the latest amyloidosis news and features for those working in and with pharma, biotech and healthcare.

AZ adds another Ionis drug in $330m kidney disease deal

AZ adds another Ionis drug in $330m kidney disease deal

volanesorsen for familial chylomicronaemia syndrome and inotersen for TTR amyloidosis in late-stage development.

Latest news

  • FDA lifts clinical hold on Sanofi’s haemophilia drug FDA lifts clinical hold on Sanofi’s haemophilia drug

    The company recently abandoned one of its gene-silencing drugs – revusiran for the rare disease hereditary ATTR amyloidosis with cardiomyopathy – although its new lead program patisiran for ATTR plus polyneuropathy remains

  • Alnylam takes off on APOLLO trial results Alnylam takes off on APOLLO trial results

    APOLLO showed that Alnylam's lead RNAi therapy patisiran is effective as a treatment for the rare disorder hereditary ATTR amyloidosis with polyneuropathy , and critically was well-tolerated by patients, sparking ... added. The 225-patient Apollo trial

  • Ionis must go it alone as GSK ditches polyneuropathy drug Ionis must go it alone as GSK ditches polyneuropathy drug

    In May Ionis reported the results of phase III trials of inotersen in familial amyloid polyneuropathy (FAP) and transthyretin (TTR) TTR amyloidosis polyneuropathy. ... Ionis said it was ‘pleased’to regain rights to the two drugs, indicating it would

  • Ionis says rare disease drug works, but safety concerns linger Ionis says rare disease drug works, but safety concerns linger

    FAP is one of a number of conditions linked to the deposition of transthyretin-related (TTR) amyloidosis, for which there are no approved therapies and a desperate need for effective drugs. ... Alnylam dropped lead amyloidosis drug revusiran last year

  • Alnylam pulls lead drug revusiran after deaths in phase III trial Alnylam pulls lead drug revusiran after deaths in phase III trial

    US biotech has yet to determine the cause of the amyloidosis and cardiomyopathy study fatalities. ... III testing to halt the progression of neuropathy in patients with hereditary ATTR amyloidosis.

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  • Tough times for pharma in Portugal Tough times for pharma in Portugal

    spondylitis. For instance, the Portuguese Association of Amyloidosis (APP) is petitioning parliament for access to Pfyzer's Vyndaqel (tafamidis). ... The drug is to be granted marketing approval by the European Medicines Agency for the treatment of

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