Major break through


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Message boards : Science (non-SETI) : Major break through

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Message 1290880 - Posted: 3 Oct 2012, 19:17:58 UTC

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.


My cousin Kerry died about 25 years ago from this dreadful disease. It has affected my Uncle to this day.

What a blessing for all those with MD. One small step closer to finding a cure.
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Message 1292530 - Posted: 7 Oct 2012, 19:51:53 UTC

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.

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Message 1292537 - Posted: 7 Oct 2012, 20:05:44 UTC

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.

That was a very brave post. Will new research help?



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Message 1292539 - Posted: 7 Oct 2012, 20:09:32 UTC - in response to Message 1292537.

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.

That was a very brave post. Will new research help?


+1

Now that is real courage.
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Message 1292826 - Posted: 8 Oct 2012, 16:06:49 UTC - in response to Message 1292539.

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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Message 1295398 - Posted: 15 Oct 2012, 0:24:25 UTC - in response to Message 1292826.

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.

Yes, It does seem like all the years of the telethons are having an effect, progress is being made, I think MD will be conquered, if this can be done, nothing will be out of reach, it will just take time and will to do the job.
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Message boards : Science (non-SETI) : Major break through

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