IMF-Pilotprojektförderung:

• “Generation and differentiation of induced pluripotent stem cells from urinary cells as a model for Fabry disease” (LE211702; 01.08.2017-31.07.2018)

•  “Specific endogenous CRISPR/CAS9-mediated transcriptional activation of α-galactosidase A to increase residual enzymatic activity in Fabry disease” (LE221704; 01.03.2018-28.02.2019)

• “Impact of neutralizing anti-drug antibodies in Fabry disease (INAF)” (LE221801; 01.01.2019-31.12.2019)

Amicus Therapeutics:

• “Fabry migalastat observational multicenter study [FAMOUS]” (NCT03135197; www.clinicaltrials.gov; 03/2017-12/2020)

Takeda:

• “Multicenter Female Fabry Study (MFFS) – impact of early ERT start on clinical manifestations in females with Fabry disease” (03/2017-12/2021)

Sanofi Genzyme:

• “Non-interventional follow-up study in patients with Fabry disease: Enzyme replacement therapy dose reduction or change of product and re-switch” (8/2017-7/2020)