WP 2 : A clinical characterization of treated SMA​

The marketing of new therapeutics has completely changed the natural history of SMA. The objectives of this WP are to study the disease course and characteristics of treated SMA patients (WP2.1), to identify new biomarkers and measures for muscular fatigue (WP2.2) and muscular atrophy (WP2.3).

WP 2.1: Natural History: Characterization of the new induced pathology of treated SMA

Vuillerot/CICpédiatrique, Servais, Freyssenet/Millet, Schaeffer, Roche, Millet, Sysnav


WP 2.2: Implementing new biomarkers for fatigue evaluation

Vuillerot/CICpédiatrique, Servais, Millet, Roche, Sysnav

WP 2.3: Cell free biomarkers for SMA

Schaeffer, Courchet, Freyssenet, Chazaud, Gache