Post by AZAFAROS B.V.

AZAFAROS B.V. has reached another important milestone: positive data from our #Phase2 (#RAINBOW) study investigating #nizubaglustat has been published in the peer-reviewed journal, Molecular Genetics and Metabolism. The data demonstrated a favourable safety profile and potential clinical benefits by reducing disease progression and seizure burdening patients with #GM2 Gangliosidosis and Niemann-Pick type C disease (#NPC). Stefano Portolano, MD, Chief Executive Officer at Azafaros, said: “These data further support the therapeutic potential of nizubaglustat to address the neuronopathic symptoms in diseases where there is an unmet medical need and reinforce our confidence as we advance our two #Phase3 studies treating patients with GM1/GM2 gangliosidoses and NPC.” Read the full story here: https://lnkd.in/eMhrR85F #rarediseases, #clinicalstudies, #biotech, #nizubaglustat, #potential, #NPC, #GM1, #GM2