On the occasion of this Rare Disease Day, Mstone Partners Healthcare and our portfolio companies are proud to stand with the ...

A veteran is taking on the ultimate challenge on the year of his 40th birthday, with a 250km marathon in his sights to raise ...

Minoryx Therapeutics and Neuraxpharm's experimental therapy for genetic neurodegenerative disorder cerebral adrenoleukodystrophy (cALD) has shown that it can slow down the progression of the disease ...

Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after the EMA started an accelerated review. If approved ...

Compiling a biorepository of pediatric patients with a range of neuroinflammatory conditions may be feasible with a longitudinal study, but obstacles to accurate data acquisition remain, according to ...

Mandatory testing of 45 genetic disorders is designed to lead to treatment before symptoms do damage.

Viking Therapeutics, Inc. (Viking) , a clinical-stage biopharmaceutical company focused on the development of novel therapies for metabolic and endocrine disorders, today announced its financial ...

Luc St-Onge, Global Head of Sales and Marketing, Minaris Regenerative Medicine; Allendale, NJ & Mountain View, CA, USA, Munich & Ottobrunn, Germany, and Yokohama, Japan; Tel: +49 89 700 9608 0 ...

POXEL SA (Euronext: POXEL - FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for ...