EMA Backs Record Number of Drugs for Rare Diseases in 2014

The European Medicines Agency (EMA) gave positive opinions on a record number of orphan medicines for rare diseases in 2014.

Of a total of 82 medicines for human use recommended in 2014, 17 are intended for the treatment of a rare disease, "providing therapies for patients who often have only few or no treatment options," the EMA notes in a news release.

For comparison, the agency recommended 11 orphan medicines in 2013, eight in 2012, and four each in 2011 and 2010.

Antibiotic breakthrough may signal the end of drug-resistant superbugs

Scientists have come across a potential game-changer in the fight against drug-resistant superbugs - a new class of antibiotic that is resistant to resistance. Not only does the new compound - which comes from soil bacteria - kill deadly superbugs like MRSA, but also - because of the way it destroys their cell wall - the pathogens will find it very difficult to mutate into resistant strains.

FDA Issues Alert On Bed Wetting Drug

The US Food and Drug Administration (FDA) has asked the manufacturers to update drug labels and prescription information on desmopressin, available in tablet and nasal spray form and used to treat bed wetting, to include new information about severe hyponatremia, a condition caused by insufficient sodium in the blood which leads to seizures and sometimes death.

Desmopressin reduces water elimination via urine, thus preventing excessive thirst, urination and dehydration brought about by a range of conditions such as types of diabetes, physical injury, surgery and other medical conditions.

Children who were being treated with intranasal forms of desmopressin for bed wetting or primary nocturnal enuresis(PNE) were "particularly susceptible to severe hyponatremia and seizures" said the FDA.

Nasal spray formulations that contain desmopressin are no longer approved for the treatment of bed wetting (PNE), said the FDA, and they should not be used in patients with hyponatremia or who have a history of it.

Diagnosing deadly cancers earlier with 'lab-on-a-chip'

A lab-on-a-chip that tests for biomarkers in exosomes – small sacs of messages released by cells, including cancer cells – shows promise as a way to diagnose deadly cancers early.

Batch-25 students of ACRI, gave a very informative, well researched and very good technical group discussion on the topic of ''TRANSLATIONAL RESEARCH".

A Systematic Review of Medical Research

*  Decide which studies to include in the review. The first and most important decision is to decide what research question will be answered. This is expressed as an aim, for example: 'To assess the effects of a new drug for a particular health problem in   certain types of people.'

 *  Decide which studies to include in the review. This will be decided in part by the tightly defined research question, but there are further 'eligibility criteria' that must be decided in advance for the inclusion or exclusion of studies. Typically, all the studies need to be of a rigorous design, such as a randomized controlled trial.

 *  Search for the studies. The sources consulted, and the search terms used to trawl them are outlined for attack - in the case of Cochrane reviews, by specially trained search coordinators. Attempts should also be made in the search to reveal unpublished studies.

 *  Select the studies and collect data from them. Data are extracted from the studies that meet the eligibility criteria set out in advance. The data relevant to the research question being investigated may have to be extracted from varied formats.

 *  Assess the risk of bias in the included studies. This is a process to ensure that all the studies reviewed are relevant and reliable. For example, was the randomization in the trial double-blinded? Or was there a risk of bias over which participants were selected for either treatment or comparison? Some studies may be of a lower quality, but they can still be included, so long as this assessment of bias is taken into account.

 *  Analyze the data and undertake meta-analyses. This is the core process of a systematic review, the main step towards synthesizing conclusions. Researchers are warned not to jump straight into this process of data analysis without first defining the aims and taking the precautions in the above steps.

 *  Address any publication bias. One of the disadvantages of systematic reviews is publication bias, which effectively means study results being cherry-picked. The risk is that a systematic review misrepresents the true effects of treatment, so this step is designed to address that concern.

 *  Present the final results of the review.

- including 'summary of findings' tables. This is the published outcome of the work used by healthcare decision-makers.

Kidney stones may increase the risk of heart disease, stroke

A kidney stone is an accumulation of waste chemicals in the kidney - calcium, oxalate, cystine, phosphate, xanthine and irate - that become a hard mass. Normally, these chemicals are passed out of the body through urine, but they can build up if there is not enough liquid to get rid of them.

Once these stones are formed, they either stay in the kidney or move down the urinary tract into the ureter. Smaller stones can be passed out the body in urine without causing major discomfort, but larger stones may become stuck. Urine can build up behind the stones, causing a lot of pain.

Study results found that patients with kidney stones are at 19% higher risk of coronary heart disease and 40% higher risk of stroke.