Author | Mathew Shanley


Tafamidis Gets Breakthrough Therapy Designation

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.

Researching Rare: Darcy Tannehill's AL Amyloidosis Journey

May 24, 2018

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.

ERTs Reduce Risk for Wheelchair Dependency in Adult Pompe Patients

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.

CALD Gene Therapy Granted Breakthrough Therapy Designation

May 23, 2018

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.