Major break through

Sareptas Muscular Dystrophy Drug Helps Patients Walk Farther .

Sarepta Therapeutics Inc.'s SRPT +176.92%treatment for a rare and terminal form of muscular dystrophy helped patients to walk about a football field farther during a six-minute test, producing "a significant clinical benefit" and more than doubling the company's market value Wednesday.

The results were from a midstage trial, but the company expects to meet with the FDA to request expedited approval in January. The trial's relatively small sample size, though, could hinder the drug's ability to get quick approval.

The drug, Eteplirsen, helps to repair a genetic mutation that results in severe muscle loss, paralysis and eventual death in young boys suffering from Duchenne Muscular Dystrophy. The disorder affects around 14,500 patients in the U.S. and about 2,000 of those have the specific mutation that Sarepta's drug targets.

Hopefully these types of break throughs can benefit suffers of other Motor Neuron Diseases.

I have recently been diagnosed with PMA ( Progressive Muscular Atrophy ) a close "cousin" disease to ALS with, unfortunately, the same prognosis...12-36 months left.

Research is ongoing and more clinical trials are in the works but it's a long, time consuming process.

agreed. I watched and didn't understand why my cousin was dying and died so many years ago.

I watched years of MDA telethons where they brought out the best and brightest Scientists describing all what they had done so far.

I can't recall the year but I remember watching when they announced the discovery of the missing Dystrophin protein.

And now a huge leap forward.

Thank you Jerry Lewis and MDA. You truly provide miracles everyday.
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End terrorism by building a school

agreed. I watched and didn't understand why my cousin was dying and died so many years ago.

I watched years of MDA telethons where they brought out the best and brightest Scientists describing all what they had done so far.

I can't recall the year but I remember watching when they announced the discovery of the missing Dystrophin protein.

And now a huge leap forward.

Thank you Jerry Lewis and MDA. You truly provide miracles everyday.