09.07.2025
1st ASMD Workshop: Focus on Neurological and Psychiatric Involvement
Experts shared clinical and research insights into neurological and psychiatric symptoms in ASMD, ranging from infantile type A to late-onset adult cases.
News
06.06.2025
INPDR-NP: Niemann-Pick registry open to all age groups
The international registry collects valuable data on ASMD and Niemann-Pick disease type C – including genetic information.
05.06.2025
Clinical trial for children with Pompe disease launched
The Zip study investigates a new combination therapy for children aged 0 to 12 years with late-onset Pompe disease.
23.05.2025
PRADO study on Tay-Sachs and Sandhoff disease
The PRADO study investigates psychiatric and neurological aspects of Tay-Sachs and Sandhoff disease. The goal is to gain a better understanding and to develop new treatment approaches.
