Scale-up of Adhesive Transdermal Drug Delivery Systems

Pharmaceutical Research -     

Myoblast transfer in duchenne muscular dystrophy

One biceps muscle of 8 patients with Duchenne muscular dystrophy was injected at 55 sites with a total of 55 million viable, purified, and contamination-free normal myoblasts (myoblast transfer). The other biceps of each patient was injected with a placebo to serve as a control. The procedure was blinded to the patients, parents, and investigators. Myoblasts derived from a biopsy specimen of the fathers were cultured and purified under strict conditions and carefully screened for microbial contamination. All patients received cyclophosphamide for immunosuppression for 6 or 12 months. No serious complications were observed after myoblast transfer, indicating that the procedure is safe. The overall therapeutic efficiency of myoblast transfer was poor as judged by the results in maximal voluntary force generation, dystrophin content of the muscle, magnetic resonance imaging of the muscle, and the lack of donor-derived DNA and dystrophin messenger RNA in the injected muscle. An improved efficiency of the take of myoblasts might be achieved by using younger cells and injecting the myoblasts with a myonecrotic agent (to increase the prevalence of regeneration) and a basal laminal fenestrating agent.     

Indirect radionuclide cystography: a sensitive technique for the detection of vesico-ureteral reflux

The detection or exclusion of vesico-ureteral reflux (VUR) has classically been by micturating cystourethrography (MCUG). Radionuclide cystography will detect VUR but fails to provide the same detailed anatomical informations as MCUG. This study allowed a comparison of indirect radionuclide cystography (IRC) and MCUG in 65 children. Renal reflux was detected by IRC in 32% of renal units, while VUR was seen in 36% by MCUG. When a comparison was made with MCUG, IRC had a sensitivity of 74.1% and a specificity of 90.5%. The markedly reduced radiation dose, avoidance of a bladder catheter plus the ability to monitor the urinary tract constantly during the entire procedure should ensure that IRC is the examination of choice in follow-up studies for VUR in all toilet-trained children.     

Lobar Pulmonary Edema Due to Mitral Regurgitation: Diagnosis by Echocardiography

A bstract One etiology of unilateral lobar pulmonary edema is mitral regurgitation. Echocardiography is able to demonstrate the retrograde flow of blood into the pulmonary veins and allows timely diagnosis and treatment planning. Correction of mitral regurgitation is followed by resolution of the radiographic abnormality.     

Accuracy of planar reaching movements

This study examined the variability in movement end points in a task in which human subjects reached to targets in different locations on a horizontal surface. The primary purpose was to determine whether patterns in the variable errors would reveal the nature and origin of the coordinate system in which the movements were planned. Six subjects moved a hand-held cursor on a digitizing tablet. Target and cursor positions were displayed on a computer screen, and vision of the hand and arm was blocked. The screen cursor was blanked during movement to prevent visual corrections. The paths of the movements were straight and thus directions were largely specified at the onset of movement. The velocity profiles were bell-shaped, and peak velocities and accelerations were scaled to target distance, implying that movement extent was also programmed in advance of the movement. The spatial distributions of movement end points were elliptical in shape. The major axes of these ellipses were systematically oriented in the direction of hand movement with respect to its initial position. This was true for both fast and slow movements, as well as for pointing movements involving rotations of the wrist joint. Using principal components analysis to compute the axes of these ellipses, we found that the eccentricity of the elliptical dispersions was uniformly greater for small than for large movements: variability along the axis of movement, representing extent variability, increased markedly but nonlinearly with distance. Variability perpendicular to the direction of movement, which results from directional errors, was generally smaller than extent variability, but it increased in proportion to the extent of the movement. Therefore, directional variability, in angular terms, was constant and independent of distance. Because the patterns of variability were similar for both slow and fast movements, as well as for movements involving different joints, we conclude that they result largely from errors in the planning process. We also argue that they cannot be simply explained as consequences of the inertial properties of the limb. Rather they provide evidence for an organizing mechanism that moves the limb along a straight path. We further conclude that reaching movements are planned in a hand-centered coordinate system, with direction and extent of hand movement as the planned parameters. Since the factors which influence directional variability are independent of those that influence extent errors, we propose that these two variables can be separately specified by the brain.