ABSTRACT
Juvenile myasthenia gravis (MG) is a rare autoimmune neuromuscular disease, often treated with anticholinesterases, corticosteroids, and immunosuppressants. However, optimal treatment durations remain unclear. This study investigated the clinical characteristics and treatment of juvenile MG, including medication duration. The administration period for all drugs, immunosuppressants, and prednisolone at doses greater than 0.35 mg/kg daily was extracted retrospectively from medical records. Nineteen participants (8 boys, 11 girls) aged 8 months to 14 years (median, 2.5 years) at onset were identified. Fourteen patients (73.7%) had ocular MG and five (26.3%) had generalized MG. Drug treatment was conducted in 18 cases; however, 7 patients did not complete the treatment. Among the patients who completed drug treatment, the duration of treatment ranged from 11 to 100 months (median, 47 months). In the six patients treated with continuous administration of prednisolone or immunosuppressants, the treatment duration ranged from 33 to 99 months (median, 56 months). No severe adverse effects requiring hospitalization were reported. The patients treated with prednisolone or immunosuppressants required at least 33 months of treatment. These results will help develop protocols for juvenile MG treatment.
ABSTRACT
The crude extracts from three species of sea anemones (Cryptodendrum adhaesivum, Heterodactyla hemprichii and Thalassianthus aster) belonging to the family Thalassianthidae exhibited potent lethality to freshwater crabs (Potamon dehaani). Regardless of the species, high and low molecular weight toxins were found in gel filtration of the crude extract. Following reverse-phase HPLC of the low molecular weight toxin fractions, one toxin (δ-TLTX-Ca1a), two toxins (δ-TLTX-Hh1a and c) and one toxin (δ-TLTX-Ta1a) were isolated from C. adhaesivum, H. hemprichii and T. aster, respectively. Based on the determined N-terminal amino acid sequences, the cDNAs encoding δ-TLTX-Ca1a, δ-TLTX-Hh1x (not assignable to either δ-TLTX-Hh1a or δ-TLTX-Hh1c) and δ-TLTX-Ta1a were successfully cloned by both 3' and 5' RACE methods. In common with the three toxins, the precursor is composed of a signal peptide (19 amino acid residues), propart (16 residues) and mature portion (49 residues), similar to those of many sea anemone peptide toxins. The deduced amino acid sequences showed that the three toxins are closely similar to one another, being all new members of the type 2 sea anemone sodium channel peptide toxin family.
