Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy.

Spinal muscular atrophy (SMA) is an autosomal recessive disease of variable severity caused by mutations in the SMN1 gene. Deficiency of the ubiquitous SMN function results in spinal cord α-motor neuron degeneration and proximal muscle weakness. Gene replacement therapy with recombinant adeno-associated viral (AAV) vectors showed therapeutic efficacy in several animal models of SMA.

Activation of multiple chemotherapeutic prodrugs by the natural enzymolome of tumour-localised probiotic bacteria.

Some chemotherapeutic drugs (prodrugs) require activation by an enzyme for efficacy. We and others have demonstrated the ability of probiotic bacteria to grow specifically within solid tumours following systemic administration, and we hypothesised that the natural enzymatic activity of these tumour-localised bacteria may be suitable for activation of certain such chemotherapeutic drugs. Several wild-type probiotic bacteria; Escherichia coli Nissle, Bifidobacterium breve, Lactococcus lactis and Lactobacillus species, were screened against a panel of popular prodrugs.

Assessing pain intensity in children with chronic pain: convergent and discriminant validity of the 0 to 10 numerical rating scale in clinical practice.

Background: In clinical practice, children are often asked to rate their pain intensity on a simple 0 to 10 numerical rating scale (NRS). Although the NRS is a well-established measure for adults, no study has yet evaluated its validity for children with chronic pain.

Objectives: To examine the convergent and discriminant validity of the NRS as it is used within regular clinical practice to document pain intensity for children with chronic pain.

Effectiveness of pamidronate for treating intractable chronic neuropathic pain: case report of two adolescents.

The objective of this study is to evaluate the effectiveness of pamidronate for the treatment of chronic neuropathic pain refractory to previous management. Intravenous pamidronate (60 mg/day for 3 days) was administered to 2 adolescents with neuropathic pain refractory to previous multidisciplinary treatments. Pain intensity, functional improvement, and adverse effects were evaluated.

Treatment of chronic intractable neuropathic pain with dronabinol: case report of two adolescents.

Objective: To evaluate the effectiveness of dronabinol for the treatment of neuropathic pain refractory to previous treatment.

Methods: We studied the response (reduction of pain intensity and functional improvement) to dronabinol (5 mg/day to 25 mg/day) in two adolescents with neuropathic pain and depression refractory to previous treatments over two and five years, respectively.

Results: Reduction in pain intensity (45%) was achieved in patient 2 and was unchanged in patient 1.