Victoria MacBean

Mitochondrial dysfunction: mechanisms and advances in therapy

Adeno-associated virus as a delivery vector for gene therapy of human diseases

P.105INCEPTUS pre-phase 1, prospective, non-interventional, natural history run-in study to evaluate subjects aged 4 years and younger with X-linked myotubular myopathy (XLMTM)

Ultrasound for the Assessment of Peripheral Skeletal Muscle Architecture in Critical Illness