A phase 2 open-label study of the safety and efficacy of weekly dosing of ATL1102 in patients with non-ambulatory Duchenne muscular dystrophy and pharmacology in mdx mice.

Background: ATL1102 is a 2'MOE gapmer antisense oligonucleotide to the CD49d alpha subunit of VLA-4, inhibiting expression of CD49d on lymphocytes, reducing survival, activation and migration to sites of inflammation. Children with DMD have dystrophin deficient muscles susceptible to contraction induced injury, which triggers the immune system, exacerbating muscle damage. CD49d is a biomarker of disease severity in DMD, with increased numbers of high CD49d expressing T cells correlating with more severe and progressive weakess, despite corticosteroid treatment.

Randomized Controlled Trial Evaluating the Use of Zoledronic Acid in Duchenne Muscular Dystrophy.

Context: Patients with glucocorticoid-dependent Duchenne muscular dystrophy (DMD) have increased fracture risk and reduced bone mineral density (BMD), often precipitating mobility loss.

Objective: To investigate use of zoledronic acid (ZA) in DMD in improving BMD.

Methods: Two arm, parallel, randomized controlled trial, set in pediatric hospitals across Australia and New Zealand.

Radiographic features of SARS in paediatric patients: a review of cases in Singapore.

Introduction: Severe acute respiratory syndrome (SARS) is a newly emerged atypical pneumonia caused by the SARS-associated coronavirus (SARS-CoV). Chest radiographic appearances have been reported as non-specific, ranging from normal to peribronchial thickening and ill-defined airspace shadowing. This study is a retrospective review of chest radiographic findings in children with suspected and probable SARS during the 2003 outbreak in Singapore.