Adverse Reactions

The Barker hypothesis, also referred to as the Developmental Origins of Adult Diseases Hypothesis, relates adverse outcomes in adults to low birth weight. A category of low birth weight individuals are those born preterm, which has been linked to abnormalities in glucose metabolism and blood pressure in childhood. Adult degenerative diseases are adversely affected by a lack of exercise. Kajantie et al from Helsinki, Finland, report that young adults (mean age 22 years) who had birth weights (Source: The Journal of Pediatrics)

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The Preventable Harm Index is a measure developed by Brilli et al at Nationwide Children's Hospital, to summarize the total number of harm events affecting pediatric patients over time. It is a simple concept and consists of adding the total number of serious adverse outcomes that are preventable. In a commentary, Brilli et al present this concept and describe how they have found it valuable in motivating their organization towards change. (Source: The Journal of Pediatrics)

Onyx Pharmaceuticals Inc. will receive $59 million upfront in a licensing deal with a Japanese company for two experimental cancer-fighting compounds, including the potential blockbuster multiple myeloma drug carfilzomib. (ONXX) (CELG) (Source: bizjournals.com Health Care:Pharmaceuticals headlines)

Onyx Pharmaceuticals Inc. will receive $59 million upfront in a licensing deal with a Japanese company for two experimental cancer-fighting compounds, including the potential blockbuster multiple myeloma drug carfilzomib. (ONXX) (CELG) (Source: bizjournals.com Health Care:Biotechnology headlines)

Canada Dry and Lipton drinks are both described in misleading terms, the agency contends in part of an ongoing clampdown on labels.The Food and Drug Administration has warned producers of two popular brands of green tea drinks not to make unauthorized nutrient claims, continuing its clampdown on misleading food labeling. (Source: Los Angeles Times - Science)

(NaturalNews) One by one, celebrities with cancer are killed off by the cancer industry and its extremely toxic (even inhumane) treatments of chemotherapy and radiation. The next potential victim is actor Michael Douglas, who recently revealed he is suffering from stage 4 throat cancer. While Douglas has the courage to admit his cancer was likely caused by his "smoking and drinking," he hasn't yet found the courage to question the toxicity of conventional cancer treatments. So he's undergoing several weeks of chemotherapy and radiation "treatments" (if you can call them that).According to his doctor, Douglas will undergo eight weeks of radiation combined with two rounds of chemotherapy. "Together, it has proven to give a good chance for a cure for the disease," says Dr Kevin Cullen, direct...

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K2M, Inc., a spinal device company developing innovative solutions for the treatment of complex spinal pathologies, announced it has received 510(k) clearance from the U.S. Food and Drug Administration (FDA) to market its new CHESAPEAKE™ Anterior-Lumbar Stabilization System, a unique interbody device designed for stabilization of the spine through an anterior approach. The system provides screw fixation through K2M's revolutionary tifix® Locking Technology, whereby each screw head forms an autogenic lock to the implant upon insertion... (Source: Health News from Medical News Today)

A brain-scanning study at the U.S. Department of Energy's (DOE) Brookhaven National Laboratory, conducted with collaborators from Stony Brook University, reveals that an oral dose of methylphenidate, commonly known as Ritalin, improves impaired brain function and enhances cognitive performance in people who are addicted to cocaine. The study - to be published in the Proceedings of the National Academy of Sciences the week of September 6, 2010 - suggests that methylphenidate, combined with cognitive interventions, may have a role in facilitating recovery from drug addiction... (Source: Health News from Medical News Today)

Canada Dry and Lipton drinks are both described in misleading terms, the agency contends in part of an ongoing clampdown on labels.The Food and Drug Administration has warned producers of two popular brands of green tea drinks not to make unauthorized nutrient claims, continuing its clampdown on misleading food labeling. (Source: L.A. Times - Health)

Conclusions: 6 MWT and 10m walk correlate well with quantitative assessment of fat infiltration. Functional measures and myometry correlate well with each other. These results open up the possibility of using these measures in a clinical trial. (Source: Neuromuscular Disorders)

The aim of this study is to establish a reliable tool of reproducible assessment of muscle strength in children affected by Duchenne Muscular Dystrophy (DMD) which will be selected for mesoangioblasts transplantation. We have developed a potential treatment for DMD based on infusion of cells (mesoangioblasts) from a healthy donor capable. The results of the current functional study will hopefully establish reliable qualitative and quantitative tool to assess results of a future cell therapy clinical trial with mesoangioblasts. This is a single centre, prospective, non-randomised, study of validation of outcome measures on 30 ambulant patients aged 5–12 years old affected by DMD including a cohort of 15 healthy aged matched males. We perform 2 days evaluation each 3 month for 1 year. Duri...

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Conclusion: Our results strongly suggest the importance to prescribe steroids for DMD patients, even after they lost ambulation. Keeping those patients in a good shape will give them a chance to be enrolled in a near future in clinical trial, for example, with exon-skipping or stem cell therapy. (Source: Neuromuscular Disorders)

Zebrafish animal models of human diseases are powerful tools for screening a large number of chemical compounds for therapeutic efficiency. The zebrafish sapje mutant and sapje like (sapc/100) are excellent models of human Duchenne Muscular Dystrophy (DMD) and each shows disturbed muscle structure and a severe reduction of birefringence (the ability to refract polarized light). The birefringence is a result of dorsal skeletal muscle deterioration and weakness that ultimately results in lethality of the most sapje mutants at 10dpf. However, a few dystrophin deficient fish can survive over 30 days. (Source: Neuromuscular Disorders)

Conclusions: Coupling genetic diagnosis with a mutation-specific therapeutic approach, ataluren is designed to enable full-length, functional protein production in patients whose disorder results from a nonsense mutation. Patients receiving low-dose ataluren experienced better 6MWD outcomes than patients receiving high-dose ataluren or placebo, potentially consistent with preclinical data suggesting that excessive exposure impedes premature stop codon readthrough. This trial comprises one of the largest prospective studies ever performed in DBMD and provides important longitudinal data in this disease. (Source: Neuromuscular Disorders)

Conclusions: There is evidence from randomised controlled trials that creatine treatment increases muscle strength, improves ADL and is well-tolerated in patients with muscular dystrophies and inflammatory myopathies. The available evidence does not support the use of creatine in metabolic myopathies. (Source: Neuromuscular Disorders)

At WMS2009, we reported that epigallocatechin-3-gallate (EGCG) given to young mdx mice in their diet or by subcutaneous (s.c.) injection reduces their serum CK activities and the numbers of lipofuscin (LF) granules, a marker of oxidative stress, per unit volume of diaphragm muscle. Concomitantly, the specific phasic twitch and tetanic tensions of triceps surae muscles are increased. These results suggest that EGCG limits the degeneration of mdx muscles. To extend our previous findings, we have investigated the effects of EGCG on mdx mice using additional assessment criteria. The EGCG doses used were 180mg/kg/day in the diet and 2.9 or 5.7mg/kg/day for s.c. injection. The mice were treated for 5 weeks beginning when they were 3-weeks-old. The integrated locomotor activities of the mice were...

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In finding new treatment for amyotrophic lateral sclerosis (ALS), one of the important therapeutic strategies is to develop inhibitors of the α-amino-3-hydroxy-5-methyl-4-isoxazole propionic acid (AMPA) receptors. This is because excessive activity of AMPA receptors, generally termed as excitotoxicity, is thought to link to the selective death of motor neurons. We are interested in developing AMPA receptor inhibitors that are both potent and water-soluble, the properties superior to all existing inhibitors. Using systematic evolution of ligands by exponential enrichment (SELEX), we have successfully identified three classes of aptamers with nanomolar affinity against AMPA receptors. In the class of competitive aptamers, we found one aptamer with an IC50 value of 30nM, rivaling any other e...

Conclusion: AVI-4658, the first PMO for DMD, was extremely well tolerated at all doses in dystrophic mice, normal mice and primates. In addition, AVI-4225, which restores dystrophin in mdx mice, also led to no adverse effects. Based on this preclinical package, and encouraging safety and dystrophin expression results from a concurrent UK clinical study, US clinical studies are anticipated. (Source: Neuromuscular Disorders)

Conclusion: Study drug well tolerated. All dosing completed. These preliminary data bode well for safe long-term administration of AVI-4658 in patients with DMD, and suggests clinically meaningful dystrophin expression can be expected following systemic administration. Preliminary laboratory data from the remaining patients at 4, 10 and 20mg/kg are due in 2Q 2010. (Source: Neuromuscular Disorders)

Distal myopathy with rimmed vacuoles or hereditary inclusion body myopathy (DMRV/hIBM) is an autosomal recessive disorder characterized by muscle atrophy, weakness that initially involves the distal muscles, and myofiber degeneration. DMRV/hIBM is secondary to mutations in the GNE gene, which encodes an essential enzyme in sialic acid biosynthesis. We recently showed that muscle atrophy and weakness were completely prevented in the DMRV/hIBM mouse after treatment with natural sugar compounds (NeuAc, ManNAc). Although this prophylactic treatment was effective, the increase of sialic acid was minimal in serum and modest in the skeletal muscles. To increase the serum and the bound sialic acid of tissues more remarkably, we screened several synthetic sugar compounds using primary human and mur...