Stockwatch: Age And Outcome Duration Cloud Innovative Therapy Reimbursement
$1m-Plus For Gene And Cellular Therapies Hard To Swallow When Patients’ Responses Differ
Gene and cellular therapies that can replace decades of expensive therapy do not come cheap. Even the initial remedy of payment by installments could be irrelevant when not all patients respond in the same way.
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Latest results show a show decline in effects, but still offers freedom from injections with almost no bleeds recorded in 13 patients.
Positive functional results from Sarepta’s SRP-9003 gene therapy in limb-girdle muscular dystrophy type 2E bodes well for a pivotal study, and for the company’s studies of other potential gene therapies in Duchenne muscular dystrophy.
Biogen’s poor third-quarter results cannot be blamed solely on the pandemic. Pipeline attrition and underperformance in most franchises pushes all the risk onto a dubious throw of the regulatory dice.