FDA Approves Duchenne Muscular Dystrophy Drug

However, a MA woman who has been purchasing the drug for her son with Duchenne muscular dystrophy (DMD), for just $1,600 per year from a British online pharmacy is not convinced with the company's argument.

For the last two decades, patients have been buying deflazacort from other countries ever since clinical trials demonstrated its effectiveness for treating Duchenne muscular dystrophy. In recent years, companies like Valeant Pharmaceuticals International and Turing Pharmaceuticals LLC have come under intense scrutiny from regulators and Congress for price gouging and abusing the rules just for profits. It can be administered to patients 5-years-old and above.

Where to buy the LG Watch Sport and Style
Buried in announcement that it would sell Google's new LG watches, Verizon has quietly announced its own Android Wear smartwatch. The one thing that seems missing is a set of extra buttons for app shortcuts, which is the latest trend with smartwatches.

Duchenne Muscular Dystrophy is caused by an absence of a protein that helps keep muscle cells intact.

Marathon Pharmaceuticals is a USA biopharmaceutical company that develops treatments for rare diseases, with a focus on patients who now have no treatment options. It is said that the condition occurs in about one in every 3,600 male infants across the globe. As the disease progresses, patients lose muscle strength, and usually end up confined to a wheelchair by the time they reach their early teens. People with the disease die in their 20s or 30s. In its response last week to the FDA's approval, the Illinois-based company said it was committed to ensuring that US patients with prescriptions have access to the drug and that the company would provide the most robust patient support programs "allowed by law to qualifying patients". However, the drug was never approved by the FDA as no companies thought it profitable enough to jump through the regulatory hoops necessary to sell it in the U.S. Patients will no longer be allowed to import the drug now that an FDA-approved product is available.

SHOOTING AT THE LOUVRE: French soldier reportedly opens fire during security scare
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The new, American, list price for this drug is $89,000 a year.

The two lawmakers say that "exorbitantly" pricing potentially life-saving medications hurts patient access and drives up prices for the entire health care sector. To compare, patients can snag the treatment for less than $1 through online pharmacies. Another study showed patients who took deflazacort lost the ability to walk later than patients who took placebo. 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.

US Coast Guard to suspend search for Canadian 'Sharkwater' filmmaker
Officials said 37-year-old Rob Stewart had gone missing Tuesday afternoon in the vicinity of Alligator Reef, off Islamorada. Despite the Coast Guard's decision to suspend the search, Stewart's parents said they aren't giving up.

Other side effects that are less common include problems with endocrine function, increased susceptibility to infection, elevation in blood pressure, risk of gastrointestinal perforation, serious skin rashes, behavioral and mood changes, decrease in the density of the bones and vision problems such as cataracts.


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