ATTR amyloidosis

Alnylam launches digital tool for AATR amyloidosis patients

Alnylam has been a pioneer in hereditary ATTR amyloidosis, getting approval in 2018 for the first drug to treat the rare disease, and has now launched a digital companion to help patients track their symptoms. The new app – called STAR – allows patients to record their symptoms and share them with healthcare professionals to …

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Alnylam Reports Completion of Patient Enrollment in P-III HELIOS-B Study for Vutrisiran to Treat ATTR Amyloidosis with Cardiomyopathy

Shots: The company has completed the enrolment in the P-III HELIOS-B study evaluates the efficacy and safety of vutrisiran (25 mg, SC, q3M, for ~ 36 mos.) vs PBO in 600 patients in a ratio (1:1) with ATTR amyloidosis with cardiomyopathy across 123 activated sites in 32 countries The 1EP of the study is to …

Alnylam Reports Completion of Patient Enrollment in P-III HELIOS-B Study for Vutrisiran to Treat ATTR Amyloidosis with Cardiomyopathy Read More »

Novo Nordisk bulks up in rare diseases with $1.2bn Prothena deal

Diabetes giant Novo Nordisk has opened a new front in its rare disease R&D, thanks to an acquisition agreement with Prothena focusing on drugs for ATTR amyloidosis. The Danish drugmaker is paying $100 million upfront for rights to antibody drug PRX004, which is heading for phase 2 trials in cardiomyopathy associated with ATTR amyloidosis, a …

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