Pr M SLAMA worked on hereditary cardiac amyloidosis with Pr David ADAMS, Pr Didier SAMUEL and Pr Dominique LE GULUDEC since 1994, within the French Referent Center for Familial Amyloid Polyneuropathy-CRMR-NNERF since 2005. We assessed and published data about liver transplantation, multimodality imaging (« bone » DPD scintigraphy, MIBG sympathetic innervation imaging, cardiac echography, cardiac MRI) for early detection of infiltrative cardiomyopathy, about prophylactic pacing for conduction disorders, and about the prognostic value of sympathetic and parasympathetic denervation.
Our group has been part of the very early assessment of new disease modifying therapies in phase III studies, focusing on the assessment of their cardiac effects:
- Tafamidis in Cardiac aTTR amyloidosis (ATTRACT B3461028)
- Antisense oligonucleotides (Inotersen-Ionis ISIS 420915-CS2)
- mRNA silencers Patisiran and Revusiran (Alnylam) (ALN-TTR-002-SC Apollo Study with Patisiran for amyloid neuropathies)