Purpose:Comprehensive review of gene replacement therapy with guidance and expert opinion on handling and administration for pharmacists.Summary:There are currently ∼2600 gene therapy clinical trials worldwide and 4 Food and Drug Administration (FDA)-approved gene therapy products available in the United States. Gene therapy and its handling are different from other drugs; however, there is a lack of guidance from the National Institutes of Health (NIH), FDA, Centers for Disease Control and Prevention (CDC), World Health Organization (WHO), and professional associations regarding their pharmaceutical application. Although the NIH stratifies the backbone biologicals of viral vectors in gene therapies into risk groups, incomplete information regarding minimization of exposure and reduction of risk exists. In the absence of defined guidance, individual institutions develop their own policies and procedures, which often differ and are often outdated. This review provides expert opinion on the role of pharmacists in institutional preparedness, as well as gene therapy handling and administration. A suggested infrastructural model for gene replacement therapy handling is described, including requisite equipment acquisition and standard operating procedure development. Personnel, patient, and caregiver education and training are discussed.Conclusion:Pharmacists have a key role in the proper handling and general management of gene replacement therapies, identifying risk level, establishing infrastructure, and developing adequate policies and protocols, particularly in the absence of consensus guidelines for the handling and transport of gene replacement therapies.

This study confirmed the beneficial effect of abobotulinumtoxinA on CD in routine clinical practice as measured by improvements in TWSTRS and PGIC. No new safety concerns were identified.

A 3-point change is the minimal clinically important change after treatment using TWSTRS as endpoint with higher cutoffs for greater baseline disease severity. For an average trial population (TWSTRS-total: 40-45), a 12-point decrease is clinically meaningful.

This study was designed to evaluate whether a weight-bearing exercise training played 3 times a week can have benefits on bone mineral density and neuromuscular function in women with a diagnosis of osteopenia. The study enrolled 22 women aged between 45 and 65, with densitometric diagnosis of osteopenia. The participants were randomly assigned to a group of exercise (n=11) and a control group (n=11). The exercise program lasted for 45 min and consisted of a combination of strength exercises that seek to cause a mechanical osteoblastic stimulus by use of gravity, body weight, fall with antigravity reaction, in combination with exercises for the improvement of balance and coordination. The outcome measures used to assess the result on bone mass are the bone Mineralometric DEXA method for femoral head-neck region and lumbar spine and biochemical markers of bone turnover (resorption and neoformation) and for the evaluation of neuromuscular function was chosen to use surface electromyography (sEMG) as an indicator of overall activity and speed activation of lumbar paravertebral muscles and of the lower limbs antigravity muscles, stabilometric analysis and 6' Walk Test. In addition each person enrolled was given EuroQol and ICF core set of osteoporosis, respectively, to assess the quality of life, as well as activity limitations and participation restrictions associated. In the exercise group, mean values and changes in average rates for the balance, muscle strength, walking ability and quality of life, mean bone mineral content and bone turnover markers, corresponding to the assessments made at 0 (before rehabilitation intervention) and Time 1 (program ended), showed a statistically significant improvement. The results of this study demonstrate that a group rehabilitation program of exercises based on gravitational load, aimed to improve muscle strength and trophism, coordination and balance, can provide advantages of unquestionable importance, not only on the slope of increase bone mass of neuromuscular function and reducing risk of falling, but on health in general.

For patients treated for cervical dystonia, switching from ONA to ABO resulted in payer and patient reimbursement cost reductions in a single U.S. private practice with outcomes assumed to be similar.

No abstract

No abstract

No abstract