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Raptor’s Drug Slows Down Muscle Loss for Patients with Huntington’s

Update Date: Feb 20, 2014 09:39 AM EST

Raptor Pharmaceutical Corporation announced that its experimental drug was effective so far in slowing down some of the debilitating symptoms of Huntington's disease. In a clinical trial, the drug was capable of slowing down patient's loss of muscle control, muscle spasms, eye and hand movements and balance after 18 months.

Huntington's disease is a neurodegenerative genetic disorder that causes the nerve cells in the brain to waste away with time. When these cells start to degenerate, the patient starts to lose basic motor cells and develop dementia. There is currently no cure for the illness.

For this trial, the researchers enlisted a total of 96 patients diagnosed with Huntington's disease. The study was designed in two parts, which were an initial 18-month phase followed by another 18-month open label treatment with the drug. The patients were randomly divided into one of two groups, which were a placebo group and the drug, RP103 group. The researchers reported that only 89 patients were able to complete the 18-month-long initial trial phase. The team analyzed data on all patients and discovered that all patients in the drug group had a Total Motor Score (TMS) that increased at a slower rate than the placebo group. The drug group's TMS progression was 32 percent slower in comparison to the placebo group.

"We are very encouraged by these trial results and we believe that the significant slowdown of loss of muscle control in these early stage patients indicates that RP103 is potentially effective at slowing the progression of Huntington's disease," said Dr. Christophe Verny, lead Investigator for the Phase 2/3 clinical trial and Chief of Neurology Department at CHU d'Angers reported by the Wall Street Journal. "We look forward to working with Raptor to develop and implement a continuing access program so that we can continue to provide RP103 to the patients who participated in the study initiated by CHU d'Angers, and avoid any treatment interruption after they finish the study."

The researchers reported that RP103 was "well tolerated." 48 out of 52 patients who took the drug and 38 out of 44 patients in the placebo group experienced at least one side effect during the 18-month phase. The researchers reported that the drug group had slightly higher incidences of gastrointestinal adverse events, such as nausea, vomiting, abdominal pain, constipation and breath odor, and headaches. Overall, seven participants dropped out of the study with six of them from the drug group.

The drug will go through more trials. Raptor is also testing the drug in a mid-stage study in children afflicted with non-alcoholic fatty liver disease.

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