Shares in Rocket Pharmaceuticals have been living up to their name, shooting up following encouraging early-stage clinical trial results from a gene therapy for a serious inherited rare hea
Delayed diagnosis, misdiagnosis, and no cure or disease modifying treatments – children living with mitochondrial disease face all the issues often associated with rare diseases.
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.
A pharma company’s core mission is to improve patient outcomes. This hinges on effectively influencing HCP clinical behaviour and driving disease education.