© 2021 MJH Life Sciences and HCPLive - Clinical news for connected physicians. All rights reserved.
© 2021 MJH Life Sciences™ and HCPLive - Clinical news for connected physicians. All rights reserved.
May 24, 2018
This morning, Pfizer announced that its investigational therapy for the treatment of patients with transthyretin (TTR) cardiomyopathy, tafamidis, received Breakthrough Therapy designation from the US FDA.
Until her death in April, Tannehill served as the Associate Professor of Education at Robert Morris University in Pittsburgh, PA. This story is about her journey to her AL Amyloidosis diagnosis.
May 23, 2018
Survey results from the IPA/Erasmus MC Pompe survey investigated whether ERT can reduce Pompe disease patients’ risk of needing either a wheelchair, respirator, or both.
Supported by positive data from an ongoing Phase 2/3 study, bluebird bio’s Lenti-D has been granted Breakthrough Therapy designation by the US FDA for the treatment of patients with cerebral adrenoleukodystrophy.