In episode 26 the pharmaphorum podcast hears from David Solomon, a 30-year life sciences industry veteran who recently took up the position of CEO at the Paris-based biopharmaceutical compa
Cure Rare Disease’s Rich Horgan discusses the importance of NAbs screening and helping rare disease patients better understand their level neutralizing antibodies as it relates to gene ther
Amanda Barrell explores how a perfect storm of changing economics, advances in technology, and the increasing volume of the patient voice is stoking change in the rare disease space.
The FDA has approved Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder.
Swedish biotech Calliditas has announced plans to file its rare disease drug Nefecon with the FDA early next year following positive results from a phase 3 clinical trial.