d*****r
发帖数: 2583 | 1 RNA一大早跌了80%..
http://www.businessweek.com/news/2013-09-20/prosensa-shares-plu
Bloomberg News
Prosensa Shares Plunge as Muscular Dystrophy Drug Fails in Trial
By Makiko Kitamura September 20, 2013
Prosensa Holding NV (RNA:US) plunged after the Dutch company’s experimental
muscular dystrophy drug failed to help patients walk better in a clinical
trial.
Prosensa sank 71 percent to $7 at 7:55 a.m. New York time in trading (RNA:US
) before U.S. exchanges opened. The company is based in Leiden, the
Ne... 阅读全帖 |
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G****a
发帖数: 10208 | 2 Dear Geisha:
Thank you for your 05/05/2011 contribution of $100.00 supporting the
Muscular Dystrophy Association. With the help of friends like you, MDA
continues to be able to provide comprehensive services through local MDA
clinics and advance its research efforts to find treatments and cures for
over 40 neuromuscular diseases. For federal income tax purposes, this letter
will serve to confirm that no goods or services were provided to you for
this donation. Please retain this letter for your ... 阅读全帖 |
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s***o
发帖数: 714 | 3 国内6岁侄儿检查出来44号基因缺失，DMD (Duchenne Muscular Dystrophy) 杜兴氏肌
肉营养不良症。
请问版上有没有做这方面研究或者是了解这个病的，能不能给点建议？不胜感激！谢谢
！！ |
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A*******s
发帖数: 9638 | 4 Muscular dystrophy at least includes the following:
Duchenne muscular dystrophy; Becker's muscular dystrophy; Emery-Dreifuss
muscular dystrophy; Limb-girdle muscular dystrophy; Amyotrophic lateral
sclerosis and Congenital muscular dystrophy
There is no effective treatment so far. |
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q********g
发帖数: 10694 | 5 观点很象这里Jenny12以前说的。从中也学习到一些分析医药股的方法。例文欣赏。
http://seekingalpha.com/article/895051-sarepta-therapeutics-loo
Sarepta Therapeutics Looks Highly Undervalued
September 28, 2012 | 37 commentsby: Sean Anderson | about: SRPT
R.S. Analytics wrote up the long case for Sarepta Therapeutics (SRPT)
earlier this month here. I agree with that thesis, and won't bore everyone
by repeating it here since R.S. did such a great job telling it already.
There are, however, a few points that I'd like to expand on.
In ... 阅读全帖 |
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g*****j
发帖数: 1211 | 6 Can that be some type of congenital muscle dystrophy?
Duchenne muscle dystrophy
Becker's muscle dystrophy
Myotonic dystrophy (less likely, late onset and positive family History)
Has he had a muscle biopsy? |
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w******y
发帖数: 4871 | 7 NOVEL THERAPIES
Gene therapy — The identification of the gene responsible for the
dystrophinopathies led to initial excitement that transfer of a functioning
dystrophin protein (whether by transplanted myoblast or direct genetic
manipulation) may provide substantial benefit to or even "cure" affected
patients. Although myoblast transfer has been attempted in humans, the
results have thus far not been encouraging [47]. However, in one patient who
received a bone marrow transplant for severe combi... 阅读全帖 |
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e****0
发帖数: 678 | 8 • leukemia
ALL AML CLL
child adult elderly
BM lymphoblasts 25%myeloblasts
lab Lymphocyte 5000, mature-appearing cells
• Osgood-schlatters disease
Adolescent male athletes
Traction apophysitis—quadriceps tendon put the traction on the
apophysis of the tibial tubercle where patellar tendon inserts.
A firm mass =heterotopic bone formation
Pain can be reproduced by extending the kne... 阅读全帖 |
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b*****d
发帖数: 61690 | 10 http://stm.sciencemag.org/content/8/331/331ed3
Funding unfunded NIH research applications
The Biomedical Research Funding Enterprise Provides The Infrastructure that
leads to new medical products that impact global health. Throughout history,
the long and successful tradition in the United States of investing in
biomedical research involves funding support by government, nonprofit
biomedical foundations, pharmaceutical and biotechnology companies, and
other private funds. However, even with the ... 阅读全帖 |
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f******I
发帖数: 769 | 11
dystrophy,
duchenne muscular dystrophy has very poor prognosis, most die in their teens
,
i'm sure mayo clinic would have the ability to test that and almost
everything else, |
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d***1
发帖数: 90 | 12 是非被确诊为BMD（Becker's muscular dystrophy） 或DMD（Duchenne Muscular
Dystrophy）。BMD相对良性一点。这个小孩多少岁，如果已经10来岁，可能是良性的。
如果小于4，5岁就经常性跌倒且大小腿增粗，有可能是恶性。建议查血碱性磷酸酶。by
the way，我有一个中药配方可缓解之。email to w*****[email protected]. |
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c*********8
发帖数: 125 | 13 Your classmate might have FSHD, facio scapulo humeral muscular dystrophy.
That is the third frequent muscular dystrophy. It is a autosomal dominant
genetic disease, so far there is no effective treatment.The life expectancy
could be normal, but need wheelchair life long.
The numbness in that disease sounds totally different from this case.
Xcontroller should be fine, he is only 33. Unless the situation is worsen
progressively, otherwise he does not need to see a doctor. |
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b*****d
发帖数: 61690 | 14 【 以下文字转载自 Faculty 讨论区 】
发信人: brihand (brihand), 信区: Faculty
标 题: NIH要和foundation合作资助unfunded proposal
发信站: BBS 未名空间站 (Fri Jun 17 13:20:56 2016, 美东)
http://stm.sciencemag.org/content/8/331/331ed3
Funding unfunded NIH research applications
The Biomedical Research Funding Enterprise Provides The Infrastructure that
leads to new medical products that impact global health. Throughout history,
the long and successful tradition in the United States of investing in
biomedical research involves funding ... 阅读全帖 |
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e****0
发帖数: 678 | 16 • Exaggerated deep tendon reflexes can be seen in lock in syndrome.
• Non bleeding varices are managed with nonselective beta-
adrenergic antagonists, such as propranolol.
• Sclerotherapy, endoscopic band ligation, and surgery are
indicated after a patient has a first episode of variceal bleeding.
• TIPS is a last resort in variceal bleeding unresponsive to
medical and endoscopic intervention.
• Ischemic colitis
CT—thickening of eth bowel ... 阅读全帖 |
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W*******n
发帖数: 4140 | 17 NYS Workers' Comp: How Alpha 3T MRI Fouls Even on The DVDs?
By Limin Wang
March 03, 2020
Introduction:
This realistic story mainly tells how a medical diagnostic imaging MRI
facility, Alpha 3T MRI (145 East 32 Street, Ground Floor, New York, NY 10016
. Phone: (212) 868-9210), gave me, an apparent workers’ compensation case (
NYS WCB# G2029240) patient but actually a victim of attempted murders, a DVD
completely blank in its content but with my information printed on the disk
skin, soon after the... 阅读全帖 |
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t**x
发帖数: 20965 | 18 Here's a (partial) list of all the pre-existing conditions the GOP bill may
not cover
感觉常见病都包了
Acne
Acromegaly
AIDS or ARC
Alzheimer's Disease
Amyotrophic Lateral Sclerosis
Anemia (Aplastic, Cooley's, Hemolytic, Mediterranean or Sickle Cell)
Anxiety
Aortic or Mitral Valve Stenosis
Arteriosclerosis
Arteritis
Asbestosis
Asthma
Bipolar disease
Cancer
Cardiomyopathy
Cerebral Palsy (infantile)
Chronic Obstructive Pulmonary Disease
Cirrhosis of the Liver
Coagulation Defects
Congestive Heart Failure
Cy... 阅读全帖 |
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w*****g
发帖数: 103 | 19 查一下是否是 Duchenne Muscular Dystrophy (DMD)？ |
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l*******x
发帖数: 14 | 20 我一个国内朋友请我帮忙，可是我什么医学不懂，请明白的同学一定指个方向啊。这是
他们的信：
我儿子在上海新华医院和北京协和医院初步诊断为进行性肌营养不良中最常见的一种类
型——杜兴氏型（简称DMD），进行性肌营养不良（progressive muscular dystrophy,
PMD）是一组原发于肌肉的遗传性变性疾病，有很多种类型，其中最常见的类型有1、杜
兴氏型（Duchenne）简称DMD，2、贝克氏型（Becker）简称BMD，这两种类型都属于X连
锁隐性遗传性肌营养不良症。杜兴氏型肌营养不良症是X染色体上的DMD基因突变，导致
缺乏抗肌萎缩蛋白（dystrophin）引起的，DMD基因突变包括79对外显子缺失、重复、
点突变。
我儿子现刚5周岁，从症状上看可能是杜兴氏型（DMD），现状况为：腓肠肌（小腿肚子
）有点大；上楼吃力要扶栏；易摔倒；蹲下起来吃力；仰卧不能直接起来，要先翻身俯
卧才能起来；跑步慢等症状。
做过的检查有1、肌电图，结果为肌源性损害不能排除；2、心肌酶谱，肌酸激酶高3600
（参考范围22-270IU/L）；3、在北京协和基础研究院做了DMD基因的79对外 |
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j*****3
发帖数: 224 | 21 Anybody hear about this and know which hospital patient can do this
treatment ? How much does it cost? Appreciate your information.
http://www.allaboutvision.com/conditions/amd-treatments.htm
FDA-Approved Macular Degeneration Treatments
Implantable telescope. In July 2010, the FDA approved a tiny, implanted
device that magnifies images onto the retina to improve central vision
damaged by AMD or Stargardt's macular dystrophy.
The Implantable Miniature Telescope (IMT) from VisionCare Ophthalmic
Te |
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c******t
发帖数: 40 | 22 我有个中学同学，他儿子得了进行性肌营养不良症（progressive muscular dystrophy
），在国内找不到医院能治，他听说美国可能相关的病有地
方治，恳请有知道的同胞告知一下，有没有治好的记录，在哪个医院能治？
万分感谢！ |
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t*******n
发帖数: 4445 | 24 Duchenne muscular dystrophy? |
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A*****a
发帖数: 52743 | 25 家族史如何？父亲以上辈的是什么情况？
听起来象cone dystrophy. 通常是显性基因控制。google这个病看能不能找到与治疗有
关的链接 |
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l*h
发帖数: 4124 | 26 is this a boy? if yes, with the CK pattern, I would first suspect Duchenne
muscular dystrophy. |
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l*h
发帖数: 4124 | 27 he will need EMG (including repetitive nerve stimulation and conduction
velocity). EMG will tell pretty well what group of diseases he has.
Definitive diagnosis can usually be achieved by muscle biopsy. Nerve biopsy
is unnecessary with this kind of presentation.
standard EMG uses fine needle electrodes. in small children, this requires
right level of sedation plus quite some experience.
if this is not possible, there is surface EMG which is non-invasive but
gathers less information.
if he has ea... 阅读全帖 |
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f****o
发帖数: 2770 | 28 Background information
Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease, is
a motor neuron disease that attacks motor nerve cells in both the brain and
spinal cord. Motor nerves control muscles that can be mentally directed,
including those for the face, mouth, arms, legs, and breathing. ALS does not
typically affect muscles that are not mentally controlled, such as external
sphincters that hold stool in the bowel and urine in the bladder, muscles
that control eye movement... 阅读全帖 |
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s******m
发帖数: 3 | 29 朋友让帮着问一下，双眼卵黄样黄斑营养不良（英语叫Best Disease 或 Vitelliform
Macular Dystrophy），国内好象没什么治疗方法，想问一下这边大家有什么保守的治疗
方法。在网上查了一下，好象现在只有在等到一定时候，用激光或者注射一种叫
bevacizumab的东西的方法。有哪位了解的能帮忙给介绍一下吗？多谢! |
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l*h
发帖数: 4124 | 32 current recommendation is to maintain moderate physical activity. don't do
intense exercise. intense exercise may cause more muscle fibers to die
early. "应该尽量少运动" is not good either. it causes loss of muscle
functions due to disuse.
I don't know about "好像是说睡觉时戴类似护膝的东西，会有好处？" |
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t*******n
发帖数: 4445 | 33 Supportive care is important for most illnesses, and for life in general.
For DMD there are advancements in recent years.
Once I came across a profile article on Pat Furlong, founder of http://www.parentprojectmd.org What she did was impressive (much more than ribbons, bake sales and demanding a cure). |
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l*h
发帖数: 4124 | 34 distrophin was identified quite some time ago. gene therapy has been under
study for some time too. one of the gene therapy studies was successful in a
variety of animals including monkeys, but the vector was not able to infect
the human cells. |
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m*****a
发帖数: 2160 | 37 从症状上看有点像Complex regional pain syndrome （也可以叫Causalgia或Reflex
Sympathetic Dystrophy Syndrome，我还真不知道中文叫什么），这个主要是临床诊断
（也就是根据症状诊断），没有决定性的检查，一些检查可能能起到辅助作用
具体科普你可以用谷歌搜这个病的英文名
治疗有多种方法，但多需要在医生指导下选择和尝试，建议再找知名点医院的神经内科
专家看看吧 |
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t*c
发帖数: 6929 | 38 看看是不是Duchenne Muscular Dystrophy，
D M D。正躺头晕可能和颈部肌肉先受损有关，但最好
叫医生再做详细检查。 |
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发帖数: 1 | 39 家里亲戚得了Fuch's dystrophy，眼睛几乎失明，需要来弯曲做角膜内皮移植 (DMEK).
请问哪位大侠知道湾区做DMEK 或者 DSAEK最好的大夫是哪一位？ 或者我应该怎么找
到好大夫啊。多谢多谢 |
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l****z
发帖数: 24 | 40 Big bless! mm要坚强，这个病治好还是有希望的！现在干细胞治疗肌肉萎缩症已有先
例，主要是对发病率最高的DMD（Duchenne muscular dystrophy，DMD）。虽然变异基
因不同，但用干细胞治疗的方法应该很类似。如果宝宝保留了脐带血，可能将来爸爸就
能用上了。如果没有，脐血干细胞排异反应比较小的，别的脐血也能用。现在大部分宝
宝出生要么保存脐血，要么捐赠。如果将来宝宝爸爸治疗需要脐血干细胞，到这儿来振
臂一呼，说不定宝宝版上的jm们真能帮上忙（不知道可不可以定向捐赠）。 |
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l****r
发帖数: 230 | 42 http://discovermagazine.com/2005/oct/sex
Last January Harvard University president Lawrence Summers hypothesized that
women may be innately less scientifically inclined than men. Not long after
the ensuing uproar, researchers announced the sequencing of the human X
chromosome. The project was hailed as a great leap forward in decoding the
differences between men and women, at least from a biological perspective.
While it did nothing to calm the maelstrom swirling around Summers, the new
understa... 阅读全帖 |
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c*******u
发帖数: 12899 | 43 ☆─────────────────────────────────────☆
karo (karo) 于 (Fri Mar 4 12:21:45 2011, 美东) 提到:
看到这样一篇文章，不知道有没有道理啊？如果是真的，我太伤心了，因为我们家都是
男宝宝，本来指望聪明的爸爸能把智商遗传给他们，现在我这个笨妈不是要害惨我的宝
宝们了。55
http://zhidao.baidu.com/question/5021375
智商的遗传基因ZT
满有意思的^^
据说决定智商的八对基因全部都是位于X 染色体上面，然后男生是XY，
X 是来自母亲，
Y 是来自父亲。
所以男生的智商全部都是来自母亲的遗传。
然后女生是X X，所以女生的智商是父亲跟母亲各有一半影响。
然后说，因为女生的智商是父亲母亲都有影响，所以会有中和的效应。
所以女生智商的分布会呈现自然分布( normaldistribution )，就是倒钟状，中间最
多，两边较少。
然后男生因为是完全只受一方影响，所以男生智商分布会呈现在偏向在两个极端。
也就是说，男生天才比较多，但是同时，蠢材之中也是男生特别多。
这个故... 阅读全帖 |
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z**b
发帖数: 192 | 44 会不会是肌肉功能方面的，如muscular dystrophy. Big bless.
来。 |
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c*******u
发帖数: 12899 | 45 ☆─────────────────────────────────────☆
jessica86 (life is short,why not make it ..able) 于 (Sat Sep 24 13:19:50 2011, 美东) 提到:
谢谢大家。很不幸，诊断是Transverse Myelitis. 现在只能希望能完全康复。
[update2]Thanks a lot, everyone. It turns out there is indeed inflammation in his spine. His attending ordered steroids and will start treatment tonight. Hopefully it works.
【update】谢谢大家。刚做完第二次MRI。脊椎也未见异常。
请大家bless。我会发100个包子。现在不够，但会买。
紧急求助：1岁半男孩，突然瘫痪，不能站立，坐。见了儿医，给转到ER。然后又转到
ICU.没有明显外伤，还有食欲。不能自主坐立。只能躺着了。右脚有反应。右手可以抬
举，但是不灵活... 阅读全帖 |
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q*********a
发帖数: 340 | 46 国内的朋友的朋友的宝宝刚刚六个星期得了肌肉萎缩（Muscular Dystrophy)，想知道
这种病美国可不可以治愈啊？问了一些朋友，好像是不治之病，朋友的朋友家觉着为了
宝宝可以豁出一切，想问问可能知道的朋友关于这个病的相关信息，知道的越多越好，
在这拜谢了。。。 |
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a*****r
发帖数: 87 | 47 你说的这个是Duchenne Muscular Dystrophy (DMD)你可以google看看 |
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w******y
发帖数: 4871 | 48 儿医那里查过duchenne muscular dystrophy吧？ |
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w******y
发帖数: 4871 | 49 儿医那里查过duchenne muscular dystrophy吧？ |
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j******u
发帖数: 41683 | 50 呵呵，刚刚看到这个，撞墙的TX可以试试，挺好，助人为乐，从而回馈自己，反正1刀
，攒攒人品也挺好的
还是看图说话
Just donate $1 to fight Muscular Dystrophy and you’ll receive a Walgreens
coupon for $3 off a $15 purchase!
呵呵，可能有的地区有的店不出，也希望白鼠TX给个报告，让大家享福
from：hip2save |
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