New data in children under 5 years with achondroplasia showed treatment with VOXZOGO was associated with improvement in tibial bowing, a common cause of pain and impaired function  Data from the largest retrospective, multi-year observational study of more than 600 children and adults with hypochondroplasia found significantly higher rates of...

Santa Cruz, Calif. – A handful of billion-dollar deals in the rare disease space highlights the uptick in Big Pharma’s investment, but it’s still extremely low compared to the money flowing to more common indications. They’re rare, they’re confounding, and R&D for therapies to address them is painfully underfunded. Rare...

LONDON, UK – Poolbeg Pharma (AIM:POLB)(OTCQB:POLBF), a biopharmaceutical company focussed on the development and commercialisation of innovative medicines targeting diseases with a high unmet medical need, announces that it has entered into an exclusive 12-month option agreement with Silk Road Therapeutics Inc, for a nominal fee, to acquire a novel...