The U.S. Food and Drug Administration approved Eteplirsen CAS# 1173755-55-9 (marketed as Exondys 51) injection, the first drug approved to treat patients with Duchenne muscular dystrophy (DMD). Exondys 51 is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent of the population with DMD.
“Patients with a particular type of Duchenne muscular dystrophy will now have access to an approved treatment for this rare and devastating disease,” said Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research. “In rare diseases, new drug development is especially challenging due to the small numbers of people affected by each disease and the lack of medical understanding of many disorders. Accelerated approval makes this drug available to patients based on initial data, but we eagerly await learning more about the efficacy of this drug through a confirmatory clinical trial that the company must conduct after approval.”
DMD is a rare genetic disorder characterized by progressive muscle deterioration and weakness. It is the most common type of muscular dystrophy. DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. The first symptoms are usually seen between three and five years of age, and worsen over time. The disease often occurs in people without a known family history of the condition and primarily affects boys, but in rare cases it can affect girls. DMD occurs in about one out of every 3,600 male infants worldwide. Read Article
A new publication is out called “May 19, 2016 – Tackling Drug-Resistant Infections Globally: final report and recommendations” and it highlights the antibiotic-resistant infections which are being seen for the 1st time in the U.S.
The review says “superbugs” could kill 10 million people each year and cost the world $100 trillion in lost economic output by 2050.
For the first time, a 49-year-old woman in Pennsylvania was infected with an E. coli which carried a gene for resistance against the drug colistin. She was successfully treated with an antibiotic. But, the Centers for Disease Control and Prevention, says “Antibiotic resistance is exploding in numbers as well as severity and the trends are alarming”. Read Article
Osteoporosis, Paget’s Disease and Alendronate
As part of the of drug family called bisphosphonates alendronate sodium works by increasing the thickness of bone by slowing down the cells that usually break down bone. This allows the cells that build bone to work more efficiently. Alendronate can help to reduce the incidence of osteoporosis-related fractures by making bones stronger.
Most patients can expect to see an increase in bone density after 3 months. Alendronate can help treat and prevent osteoporosis as long as it is taken consistently. Alendronate controls but does not cure osteoporosis and Paget’s disease of bone, but instead slows down the progression of these conditions.
Short-term use of Alendronate
In results from the Fracture Intervention Trial Long-term Extension (FLEX) study published in 2006 suggests that some women can eventually stop or take a break from taking Alendronate. That study included women who had taken Alendronate for at least five years. The participants were randomly assigned to continue the drug or switch to a placebo for five more years. Individuals who discontinued use showed a slow decline in bone density and a moderate increase in the risk for spine fractures. The rate of hip fracture, a far more serious injury, was the same in the both groups. Therefore, Alendronate treatment significantly reduced the incidence of injury and hospitalizations. Read Article
The drug Acamprosate, sold as Campral®, is used (in combination with counseling) to help people recovering from alcoholism to avoid drinking alcohol again. It reduces alcohol consumption in animal models of alcohol addiction.
When a person has been drinking large amounts of alcohol for a long time, it changes the way their brain works. The changes that occur involve two specific neurotransmitters called glutamate and GABA (gamma-aminobutyric acid). In a healthy brain, glutamate is released in order to encourage more rapid communication between nerve cells called neurons, while GABA is released to slow communication between the brain’s neurons. When alcohol abuse happens, the levels of neuron-exciting glutamate rise, while levels of neuron-calming GABA falls. This leads to overstimulation of the brain and an increased urge for alcohol.
Acamprosate is considered a safe and well-tolerated treatment for most patients with alcoholism. It also appears to help patients remain abstinent. This compound is a featured product for neuroscience research. In a recent article on the Journal of American Medical Association discussed a study on Acamprosate to determine its significance in helping people recovering from alcoholism. It included a systematic review and meta-analysis of the benefits for adults with alcohol use disorders. In the final analysis, Acamprosate was shown to be effective in helping people reduce their return to abusing alcohol.
Acamprosate works by helping restore normal brain function of people who have consumed large amounts of alcohol. It works by restoring the chemical balance in the brain in an individual who has recently stopped drinking.
Acamprosate and R&D
Acamprosate does not prevent the withdrawal symptoms that a recovering individual may experience when they stop drinking alcohol. Acamprosate helps to prevent a person from drinking alcohol only as long as they take it. A benefit of Acamprosate is that it will not cause an unpleasant reaction if alcohol is consumed during treatment.
LGM Pharma can assist clients as a supplier/distributor of the Acamprosate CAS# 77337-76-9. Clients can be assured of quality API products and continuous support throughout the R&D process. We specialize in supplying our customers with a wide range of APIs supported by integrated technical capabilities and access to complete regulatory DMF documentation, suitable for various R&D stages through commercial formulation production, to various global pharmaceutical companies and academic research institutes.
Insulin Glargine CAS# 160337-95-1:
Insulin Glargine is basically used as a treatment for Type-1 diabetes, a condition in which the body is not able to produce enough insulin, and as a result, is unable to control the amount of sugar in the blood stream. It is also used for the treatment of Type-2 diabetes where the body is unable to use insulin normally. Insulin Glargine is a synthetic version of insulin which is long lasting and works by replacing the insulin which is naturally produced by the body in order to move sugar from the blood to other parts of the body, where it is stored and used for energy.
In recent clinical trials, a fixed ratio combination of Insulin Glargine along with Lixisenatide, which is a GLP-1 RA was shown to have superior reduction in the average blood glucose levels as compared to past clinical trials. Overall, the fixed ratio combination showed to have a safety profile which reflected those of insulin Glargine and Lixisentide. According to the researchers, the meeting of the primary objectives of this clinical trial showcases the clinical value of Insulin Glargine. The study is said to be completed by Q3 of 2015. Read Article