Elevidys is the first-ever gene therapy for DMD, a neuromuscular disease characterized by progressive muscle weakness and atrophy that strikes primarily young boys
Details of the registries and codes used for cancer diagnoses, drug exposure and covariates are provided in electronic supplementary material (ESM) Methods and ESM Tables 13
Seek emergency care (call 999) if you experience sudden severe breathlessness, chest pain, facial swelling, wheeze, or severe abdominal pain
Tumor cells activate multiple carcinogenic pathways, leading to increased expression of transporters and metabolism-related enzymes, which in turn results in enhanced uptake of glutamine by tumor cells and massive consumption of glutamine in the TME [7, 23]
Example: How Dosing Works Program 1: ~2.5 mg weekly / 10 mg vial ~4 weeks Program 2: ~5 mg weekly / 20 mg vial ~4 weeks Program 3: ~7.5 mg weekly / 30 mg vial ~4 weeks Program 4: ~10 mg weekly / 40 mg vial ~4 weeks Program 5: ~12.5 mg weekly / 60 mg vial ~4 weeks Starter: ~2.5 mg weekly / 10 mg vial ~4 weeks Maintenance: ~5 mg weekly / 40 mg vial ~8 weeks Examples only
27 The authors documented that erastin triggered mitochondria-independent oxidative stress causing cell death, which could be prevented by iron chelation
