• Latest Posts

Selecta and Astellas sign LOPD treatment agreement 

NMD Pharma cleared for spinal muscular atrophy trial

Metrion and KCNC1 Foundation announce collaboration to identify therapeutic

New mitochondria study could pave way for disease treatments

Enzymes could be key to understanding ‘origin of DNA errors’

Aro Biotherapeutics gets FDA orphan drug designation for treatment of Pompe Disease


Trial for adults with late onset Pompe disease halted due to serious adverse event