Safety and Efficacy of Apitegromab in Patients With Spinal Muscular Atrophy Types 2 and 3: The Phase 2 TOPAZ Study.

Background And Objectives: Currently approved therapies for spinal muscular atrophy (SMA) reverse the degenerative course, leading to better functional outcome, but they do not address the impairment arising from preexisting neurodegeneration. Apitegromab, an investigational, fully human monoclonal antibody, inhibits activation of myostatin (a negative regulator of skeletal muscle growth), thereby preserving muscle mass. The phase 2 TOPAZ trial assessed the safety and efficacy of apitegromab in individuals with later-onset type 2 and type 3 SMA.

An Epithelioid Hemangioma Camouflaged As Radial Aneurysm.

Epithelioid hemangioma (EH) is an uncommon benign vascular lesion usually present as subcutaneous nodules in the head and neck area. Sometimes, these lesions can occur in the peripheral arteries, and when they do, they can be mistaken for aneurysmal dilatations of that respective vessel. We report a case of a 43-year-old male who underwent surgical recession of a radial aneurysm, which after anatomopathological examination, revealed an EH.

Advances and limitations for the treatment of spinal muscular atrophy.

Spinal muscular atrophy (5q-SMA; SMA), a genetic neuromuscular condition affecting spinal motor neurons, is caused by defects in both copies of the SMN1 gene that produces survival motor neuron (SMN) protein. The highly homologous SMN2 gene primarily expresses a rapidly degraded isoform of SMN protein that causes anterior horn cell degeneration, progressive motor neuron loss, skeletal muscle atrophy and weakness. Severe cases result in limited mobility and ventilatory insufficiency.

Gestational age at birth and morbidity, mortality, and growth in the first 4 years of life: findings from three birth cohorts in Southern Brazil.

Background: We assessed anthropometric status, breastfeeding duration, morbidity, and mortality outcomes during the first four years of life according to gestational age, in three population-based birth cohorts in the city of Pelotas, Southern Brazil.

Methods: Total breastfeeding duration, neonatal mortality, infant morbidity and mortality, and anthropometric measures taken at 12 and 48 months were evaluated in children of different gestational ages born in 1982, 1993 and 2004 in Southern Brazil.

Results: Babies born <34 weeks of gestation and those born between 34-36 weeks presented increased morbidity and mortality, were breastfed for shorter periods, and were more likely to be undernourished at 12 months of life, in comparison with the 39-41 weeks group.

Posterior fontanelle sonography: an acoustic window into the neonatal brain.

Background And Purpose: Sonographic brain studies are classically performed through the anterior fontanelle, but visualization of posterior supratentorial and infratentorial structures is poor with this approach. Posterior fontanelle sonography is recommended for better assessment of these structures. Our purpose was 1) to determine whether sonography of the brain through the posterior fontanelle (PF) improves visualization of brain lesions when added to the routine anterior fontanelle (AF) approach and 2) to describe standardized PF coronal and sagittal sections.

Epidermal growth factor receptor is a predictor of tumor response in locally advanced rectal cancer patients treated with preoperative radiotherapy.

Purpose: Epidermal growth factor receptor (EGFR) expression is observed in 50%-70% of colorectal carcinomas and is associated with poor prognosis. The aim of this study was to determine the EGFR expression rate in locally advanced rectal cancer and to analyze whether EGFR expression predicts tumor response to preoperative radiotherapy.

Methods And Materials: Between December 1997 and October 2000, 45 patients were included.

[Antimicrobial use in Spanish hospitals (EPINE, 1990-1999)].

Background: Monitoring of antimicrobial use and knowledge of prescription habits are some of the strategies recommended to contain the resistance to antimicrobials in hospitals.

Method: We analyzed a series of 10 annual studies of prevalence during the period 1990-1999 (EPINE project) in Spanish hospitals. Estimates of antimicrobial use were calculated as the percent distribution of every antimicrobial related to the overall antimicrobials prescribed.

[Etiology of hospital-acquired infections in Spanish hospitals (EPINE, 1990-1999)].

Background: Clinical and demographic characteristics of patients, their interaction with pathogens and antimicrobial therapies are prompting changes in the epidemiology of hospital-acquired infections (HI). The knowledge of the etiology of hospital-acquired infections is valuable for the treatment of infected patients and for the prevention of HI.

Patients And Method: We analyzed a series of 10 annual prevalence studies during the period 1990-1999 (EPINE project) in Spanish hospitals.