THE 5-YEAR EU PROJECT SCREEN4CARE ON GENETIC NEWBORN SCREENING – SELECTION OF TREATABLE DISEASES

A multicentre, prospective study comparing autoantibody diagnostic assays in myasthenia gravis

Preclinical assessment of the effects of the growth hormone secretagogue JMV2894 in the D2-mdx mouse model of Duchenne muscular dystrophy

Countermeasures for sarcopenia: effects of BCAA-based formulations on muscle function, histomorphology, and calcium homeostasis in aged mice.

Natural history of distal and myofibrillar myopathies assessed by clinical and technological outcome measures (Dista-Myo): longitudinal results

Need for tube feeding in SMA type I patients treated with disease modifying therapies: prognostic factors

Functional characterization of the Nav1.4 sodium channel mutation, p. L689F, found in a young boy suffering from myotonia permanens.