MYH2-associated myopathy caused by novel compound heterozygous mutations: a case report and literature review
Morales PN, Coons AN, Koopman AJ, Patel S, Chase PB, Parvatiyar MS, et al. Post-translational modifications of vertebrate…
Machine learning in Alzheimer’s disease genetics
School of Medicine, Cardiff University, Cardiff, UK Matthew Bracher-Smith, Julie Williams, Nicola Denning, Alun Meggy & Valentina Escott-Price Centre for Neuropsychiatric Genetics and…
AAV mini-dystrophin gene therapy for Duchenne muscular dystrophy: a phase 1b trial
Study design and oversight This ongoing, nonrandomized, open-label, ascending-dose, phase 1b study (ClinicalTrials.gov registration no. NCT03362502) was initiated…