Presymptomatic treatment with risdiplam was associated with improved motor development and survival without permanent ventilation in infants with spinal muscular atrophy (SMA), according to 24-month results from the phase 2 RAINBOWFISH trial published in the New England Journal of Medicine.
Nearly 1 in 10 individuals with myalgic encephalomyelitis/chronic fatigue syndrome reported voice or speech changes in a recent survey, despite not being directly asked about these symptoms.
Dysregulated Ca²⁺ signaling due to ANO3 mutations impairs K⁺ channel activation in striatal neurons, offering new insights into the pathophysiology of dystonia.
A patient presents with sudden-onset right-sided otalgia accompanied by vertigo, emesis, and auditory impairment. Your otoscope reveals an unexpected finding. What's your diagnosis?