Marathon Pharmaceutical's deflazacort, a steroid, aims to improve the lives of patients with Duchenne muscular dystrophy by improving muscle function, potentially allowing them to walk, feed themselves and control their wheelchairs longer. Because the FDA has approved a drug to treat the disease, US consumers won't be legally able to acquire it overseas at the cheaper price anymore. Emflaza is a corticosteroid that works by decreasing inflammation and reducing the activity of the immune system. 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. Instead, the drug will carry a list price of $89,000 a year, though the company said it will provide a "robust" assistance program for patients who are not covered by insurance.
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The visits were organised at the request of the refugees, many of whom are Syrians fleeing the civil war in the country. The city's main bus station was shut down, trains in the area were halted and churches cancelled their Sunday services.
In a Thursday press release, the Food and Drug Administration (FDA) granted approval for a drug by Marathon Pharmaceuticals, a USA company focused exclusively on the development of new treatments for rare diseases. The cost of treatment with importing the drug from Europe is almost $1,200 per year. This is the first FDA approval of any corticosteroid to treat DMD and the first approval of deflazacort for any use in the United States. Life expectancy and disease severity vary but many patients usually succumb to DMD in their 20s or 30s. This particular drug is meant to treat a disease that impacts about 12,000 boys each year in the United States.
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Herpetologist Josh Zalabak offered one possible explanation for the unusual coloring of the reptile. The unique reptile was discovered on the banks of a retention pond in Tanner Plantation in Hanahan.
The FDA approved Emflaza for all DMD patients aged 5 and older. It is a practice that has prompted congressional investigations for other companies in the past, most notably, Turing Pharmaceuticals, the firm formerly run by one time hedge-fund manager Martin Shkreli, who took a drug used to treat AIDS patients, and increased 5,000 percent from $13.50 a pill to $750. An overall stability in average muscle strength was maintained through the end of study at week 52 in the deflazacort-treated patients.
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Thomas Cook is raising its prices this year, it will cost on average 9 per cent more to fly away on holiday this summer. Partly because of the prevailing high prices in Spain, bookings to Greece for summer 2017 are up by almost half.
Similar results were observed in another trial that lasted 104 weeks with 29 male patients but this experiment also showed that participants on deflazacort seemed to lose walking ability later than those given placebo. This is the 9th time that the FDA grants the pediatric disease priority voucher. More importantly, the drug results in less severe side effects typically experienced when using corticosteroids, scuh as bone mass loss, weight gain, behavioral issues, and glucose intolerance. Other side effects include elevated blood pressure, mood changes, infection, and serious skin rashes.