Single-center long-term follow-up of 18 patients with Spinal Muscular Atrophy (SMA) treated with risdiplam

SYNE-1 and SYNE-2 mutations: expanding the genotype and phenotype spectrum of nesprinopathies

Exploring the Effects of Risdiplam-like Compound on Spinal Muscular Atrophy Using a 3D Stem Cell-Derived Spinal Cord Model

Early real-life experience with Efgartigimod in AChR-seropositive generalized myasthenia gravis

SEPN1-RELATED MYOPATHY WITH ACUTE RESPIRATORY ONSET IN MIDDLE AGE: A CLINICAL AND GENETIC STUDY

GAIT PARAMETERS / COGNITIVE FUNCTION CORRELATION IN A COHORT OF PATIENTS WITH MYOTONIC DYSTROPHY TYPE 1: A SINGLE-CENTER SENSOR-BASED GAIT ANALYSIS

ANTI-SRP IMMUNE-MEDIATED NECROTIZING MYOPATHY IN A PATIENT WITH ANTI-ACHR MYASTHENIA GRAVIS AND THYMOMA.

Anti-FcRn treatment for generalized Myasthenia Gravis: a real word experience with Efgartigimod in AChR-Ab positive patients.

Preferential binding to adult or fetal acetylcholine receptor isoform as a promising predictive biomarker in myasthenia gravis