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The new deal struck by the NHS will enable provision of a life-saving treatment for babies and young children who suffer with a rare and fatal genetic disease, metachromatic leukodystrophy (MLD). The revolutionary gene therapy treatment, known by its brand name Libmeldy, is used to treat MLD, which causes severe damage to the child's nervous system and organs, leading to a life expectancy of just five to eight years. Having a reported list price of more than £2.8 million, it is the most expensive drug in the world, but can now be offered to young patients on the NHS in England after the health service negotiated a significant confidential discount. the drug works by removing the patient's stem cells and replacing the faulty gene that causes MLD before then re-injecting the treated cells into the patient. The most common form of MLD usually develops in babies younger than 30 months and can cause loss of sight, speech and hearing, as well as difficulty moving, brain impairment, seizures, and eventually death.

An oral drug combination by Novartis showed promise in treating a subgroup of patients suffering from a common childhood brain cancer in a trial. In the mid-stage trial, 47 per cent of the patients that were given the two drugs Tafinlar and Mekinist saw their tumours shrink, far above a rate of 11 per cent in a comparative group of participants on standard chemotherapy, the drugmaker said on Monday, June 6. The participants, aged one to 17 years, were suffering from low-grade gliomas (LGG), the most common childhood brain cancer. The trial only included those who were found to have a mutation known as BRAF V600, a genetic contributor in about 15 per cent to 20 per cent of paediatric LGG cases. Among further results of the trial with 110 participants, the median time without disease progression was 20.1 months for those given the Novartis drug combo, compared to 7.4 months on chemotherapy. The new oral treatment candidate also caused less severe side effects than burdensome chemotherapy.

The National Institute for Health and Care Excellence (NICE)'s independent committee has called for more research to ensure liposuction for treatment of chronic lipoedema is safe and effective enough for use on the NHS. Lipoedema is more prevalent in women and very rarely affects men. It is characterised by an abnormal, usually symmetrical, accumulation of fat in the legs, hips, buttocks, and occasionally arms. It is a separate condition to obesity and lymphoedema. The cause of lipoedema is unknown, but hormonal changes, weight gain and genetics are thought to be involved. The size and shape of legs, and the resultant mobility issues and pain, can have a profoundly negative effect on quality of life, and physical and mental health. The interventional procedures advisory committee reviewed evidence from several sources, including eight before-and-after studies. The committee was informed that several different liposuction techniques are used and that they may have different safety and efficacy profiles.

The European Union's health regulator on Friday said it had recommended granting a conditional marketing authorisation for a gene therapy by Australian drugmaker CSL Ltd and partner uniQure N.V. , to treat haemophilia B, a rare bleeding disorder which is caused by genetic anomalies. About one in 40,000 people are affected by the inherited disorder, caused by a gene mutation that hampers the body's ability to make clotting protein factor IX. If approved, the treatment, branded Hemgenix, will be the first gene therapy in the European Union for the condition that is usually treated by regular injections of factor IX, the European Medicines Agency said. CSL had acquired exclusive global rights to Hemgenix in May last year from uniQure N.V.

One type of bacteria found in the gut may contribute to the development of Type 2 diabetes, while another may protect from the disease, according to early results from an ongoing, prospective study led by investigators at Cedars-Sinai. The study, published in the peer-reviewed journal Diabetes, found people with higher levels of a bacterium called Coprococcus tended to have higher insulin sensitivity, while those whose microbiomes had higher levels of the bacterium Flavonifractor tended to have lower insulin sensitivity. Mark Goodarzi, the director of the Endocrine Genetics Laboratory at Cedars-Sinai in Los Angeles, California, is leading an ongoing study that is following and observing people at risk for diabetes to learn whether those with lower levels of these bacteria develop the disease. "The big question we're hoping to address is: Did the microbiome differences cause diabetes, or did diabetes cause the microbiome differences?" said Goodarzi, who is the senior author of the study and principal investigator of the multicenter study called Microbiome and Insulin Longitudinal Evaluation Study (MILES).

After receiving approval from the National Institute for Health and Care Excellence (NICE), the NHS is preparing to roll out a new combination treatment for brain tumours in children and young people in England. Dabrafenib with trametinib would be the first ever targeted treatment for children (aged 1-17) with gliomas that have a specific genetic mutation, the health service said. The treatment, which can be taken at home, has been found to slow the progression of the disease by over threefold compared to standard chemotherapy in children with low-grade gliomas that have a BRAF V600E mutation. Clinical trials have shown that it causes fewer side-effects than chemotherapy. NICE approved the combination treatment on Wednesday 24 April. It will be available on the NHS in the coming months.

Know about the mechanism of DNA Translation in prokaryotes and eukaryotes and various factors involved.

Psoriasis is a chronic skin condition that accelerates the rate at which your skin cells grow. An effective method to treat psoriasis is phototherapy or light therapy.

Psoriasis is an auto-immune illness that affects almost three percent of the world's population. People who suffer from psoriasis experience a great deal of skin irritation and discomfort.