The European Medicines Agency has backed expanding Namuscla to address muscle rigidity in children and adolescents with rare ...
Background A young adult female presented with syncope and periodic weakness. A 12-lead electrocardiogram showed frequent premature ventricular contractions and prolonged QU interval. Repetitive runs ...
Diseases of unstable repeat expansion are a diverse group of disorders that are caused by the expansion of trinucleotide, tetranucleotide, or pentanucleotide repeat sequences. The position of the ...