Registry Gangliosidoses

key information

study id #: NCT04624789

condition: Gangliosidoses, GM1 Gangliosidosis, Morquio B Disease, Sialidosis, Galactosialidosis, Gm2-Gangliosidosis, Variant B1, Tay-Sachs Disease, Sandhoff Disease, GM2 Activator Deficiency

status: Recruiting


The clinical project “Eight At One Stroke: Attention Gangliosidoses” represents a clinical registry for recording the clinical manifestation and the disease progression of gangliosidoses. The intention of this project is to better understand the manifestation and progression of gangliosidoses and to raise awareness of these disorders in the public health service. The patients or their families, respectively, will be integrated in the study in order to measure Patient Outcome and to objectify the psychosocial burden for the patient and his family. The study has a retrospective and a prospective part. It is planned to transfer the data of the study into a continuous registry.


last updated: February 26, 2022