Genervon GM6 Offers a Sliver of Hope for ALS Patients – PLEASE SHARE
dhuntley
By dhuntley · January 14, 2015 at 1:20 pm · 49 replies
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Finally, 75 years after Lou Gehrig’s famous speech, there may be some hope for People with ALS (PALS). A small bio-tech firm in Pasadena, Calif., has recently completed a Phase II trial to successfully show safety of a new drug, called GM604 (or GM6). Though the trial was designed only to show safety, much to their surprise (because of the small sample size), the results suggest GM604 will be effective in treating ALS. Seven of the eight patients treated with GM604 saw their progression slow OR STOP! Stopping progression of ALS is unprecedented, finally giving PALS some hope. In addition, GM604 was given, on a compassionate-use basis, to an additional person who has had ALS for nine years and has been a quadriplegic and on a ventilator for seven years. The result was an improvement in both his speech and ability to swallow. These results are described in more detail at
Genervon GM6带来希望的ALS患者银 - 请您分享
亨特利
通过亨特利·2015年1月14日1:20点·49回复
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最后，卢伽雷著名演讲75年后，可能会有一些希望患有ALS（PALS）。在加利福尼亚州帕萨迪纳的小生物科技公司，最近完成了II期临床试验，成功展现出新的药物的安全性，被称为GM604（或GM6）。虽然试验设计为只显示安全，这让他们吃惊的是（因为小样本），结果表明GM604将有效地治疗ALS。八例GM604治疗七看到他们的进展缓慢或停止！停止ALS的进展是前所未有的，终于让PALS一些希望。此外，GM604被赋予，在一个富有同情心使用的基础上，谁拥有了ALS九年，一直是四肢瘫痪和呼吸机七年额外的人。其结果是在这两个讲话和吞咽能力的提高。这些结果更详细地描述在
The hurdle to overcome before Genervon is allowed to make GM604 to PALS is the FDA. Under normal circumstances GM604 would go to a much larger phase 3 trial to test for efficacy before applying for approval by the FDA. That process will take 3+ years to complete. Roughly 5000 people are diagnosed with ALS every year in the US alone, and the median life expectancy is 3 to 5 years from diagnosis. That means that, in the 3+ years required to complete a phase 3 study, more than 15,000 people will be condemned to die from ALS. I am one of those people. I completed an Ironman Triathlon 1 ½ years ago and was diagnosed with ALS soon after. In the time since diagnosis I have lost the ability to run, walk, stand, speak intelligibly, eat solid foods, as well as losing half of my breathing capacity. Without access to GM604 I will pass in the next 6 to 12 months.
Genervon之前克服的障碍是可以使GM604到PALS是FDA。在正常情况下GM604会去一个更大的3期临床试验申请批准前，FDA以测试效果。这个过程将需要3年以上才能完成。大约有5000人在美国，每年诊断出患有ALS独自一人，并且平均预期寿命为3至5年后诊断。这意味着，在完成第三阶段的研究需要3年以上，超过15000人将受到谴责，从ALS死亡。我的那些人之一。我完成了铁人三项1年半前，很快就被后确诊为ALS。自从确诊的时候，我已经失去了跑步，走路，站立的能力，说话也听得清，吃固体食物，以及失去了一半我的呼吸能力。如果没有获得GM604我会把在未来6至12个月。
Because ALS is 100% fatal, and because the phase 2 trial results are so promising, Genervon is meeting with the FDA in February to request Conditional Approval to make GM604 available to PALS, with post-marketing analysis of the results. The risk to PALS is small…..if GM604 works our progression is slowed or stopped. If it doesn’t work, we die. But if we are not provided access to GM604 there is a 100% certainty that those of us that are progressing at an average or fast rate will die before a phase 3 trial is completed.
WHAT CAN YOU DO?
1. Go to and sign the petition asking the FDA for conditional approval of GM6.
2. Go to and submit a comment to the FDA supporting conditional approval of Genervon GM6 for people with diagnosed ALS.
3. Write your congressperson and senator asking them to urge the FDA to grant conditional approval.
4. Share this post on every social media you use. This message needs to go viral
因为ALS是100％致命的，因为在第2阶段的试验结果是这样希望的，Genervon是与FDA开会二月要求条件批准使GM604可供PALS，对结果的上市后的分析。以PALS风险小... ..如果GM604我们的工作进展减慢或停止。如果它不工作，我们死。但是，如果我们不提供访问GM604是有100％的把握，我们这些正在取得进展，平均或快速率3期临床试验完成之前就会死亡。
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