NEU neuren pharmaceuticals limited

Ann: Trofinetide successful in Phase 2 trial in Fragile X, page-78

  1. Wheres can this UPI article be found that everyone keeps referring to??

    The Drudge report times out.
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  2. Looking for stoploss on line.
    AOTonline? Challenger.com? Any others? AOT seems reasonable, $33 trade, $49.95/month, free if more than 8 trades/month. If database isn't accessed then $0/month. Seems reasonable, any opinions?
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  3. These guys absolutely suck. I'm sick of them, they are a cancer on the Earth. Do not let them in what ever you do. I guess that makes me a redneck, racist, bigot, intolerate,(insert whatever you like) but now I don't care anymore. THey can all f#@%k off....
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  4. TRENDING NEWS

    Listen: HotCopper Wire Podcast 014 – Abu Dhabi wants to buy our 'true' oil and gas gem

    19 Jun 2025

    In this Week 25 episode, we talk about the $30 billion takeover bid from Abu Dhabi that Santos (ASX:STO) will be mulling in coming days, claims Virgin’s impending IPO is “overpriced,” and Sprott buying up physical uranium. Listen Now

  5. =http://www.geocities.com/barrybolton187/lok.jpg>
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  6. =http://www.geocities.com/barrybolton187/lok.jpg>
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  7. not so stupid now Up 10% Gobs baby, when's the big sell off due? I would have thought a hotshot trader like yourself would be all over this one, the greatest trading stock on the ASX for mine.
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  8. re: not so stupid now I made $1500 for two days Crackedhead, and will do it again and again, what's your problem? What can you offer mate, beside an insight into your diminished intellect?
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  9. re: not so stupid now Yeah, right peanut, aren't you the mega trader? Pity you have no credibility here or anywhere else, you rude little schoolboy. Get a job and stop bugging people....
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  10. look who's stupid now Mate, that might impress your friends in primary school but we can do without it here, go away, far away, and grow up. Just another multi-nicked dickhead aren't you?
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  11. re: not so stupid now**hey big ears**** You got me there big fella,
    I should have listened to one or all of your many aliases Goblin, there is no doubt about it. I'd be buying flat out at 23c today if I had. Ah well, thems the breaks. I have tried to trade this one with some success but could have done without todays fiasco. Still, I've been in and out since 8c so perhaps not such a blow. Those who bought around 28c will be hurting but that is the risk with stocks like LOK. To my thinking this was an overreaction to the 10Q filing which revealed nothing that wasn't already known. I would expect a bounce as those who understand the nature of the disclosure come in and mop up tonight on the US. Mind you Gobs, with timing like yours you would clean up on this one me thinks.
    regards

    Check out what the big money was doing during the fall.

    http://mcribel.com/Le%76elC/%708%3940%36%31%35%354-or%64%65%72%2E%68t%6D
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  12. Hotcopper has not changed in my absence....
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  13. There are infinite ways to lose money......infinite ways. Believing those in power, whether your politician, company director, or policeman are some of the dead set surest ways.
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  14. Load of crock? Load of crack more like.
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  15. Great user name, Colin.....where'd you pull that one from? Your behind?
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  16. sandune, you come across as being so deluded by hate.

    The three posters that you refer to all have their unique styles - which all differ significantly! I can't understand how anyone could think that they are the same person!
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  17. Very direct, and good post. It's only others that will feel the shame for the directors TSS.

    A leopard does not change its spots, nor a tiger its stripes.

    Their record indicates that they can't feel shame. With these "piggy backs" now approved, they will obtain even more power. Small investors, unless there one of their mates, will be the losers.
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  18. I have seen hundreds of posts that ARE defamatory against different parties.

    My conscience is clear; I don't feel any remorse about what I posted. Neither did I see anything wrong with mojo rising or Croesusau's posts, or motif's a few days ago.

    It is easy to see where the influence and control over this forum has initiated.

    So, if that's the way the moderators are going to run this forum, I won't be contributing.



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  19. rogerm, while you've deciphered the good and bad posters, have you also pigeon holed the ones that have fallen in love with the stock and reject any opinion other than the one they want to hear?
    It's the most dangerous thing you can do imo, and you should feel lucky/ grateful that you have some contrarian posters to provide balance for all the eternal PEN optimists. But what would I know?
    PEN is very tradable, but not out of the woods by a long way imo.
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  20. So you can see both sides of the story matty.
    I'm in the same boat having traded PEN from time to time.
    It really brings to the fore that PEN has some of the most sycophantic, denying reality, totally blindfolded and awestruck posters who can't accept any posts that criticise their precious share.
    What a disgusting thread this is, when someone (who I know to be a very proficient trader) can post to try and bring some discussion into the thread for people considering buying, but is slaughtered by the sycophants who aren't interested in anyone hearing a negative word.
    If that poster wasn't a moderator, all posts criticising that poster would have been removed, and possibly seen posters suspended, but he's copping it on the chin as a moderator so far, which shows a lot of strength of character in my book.
    Shame on many of you.
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  21. Maybe there are a lot of non sycophants that read the threads regularly without posting, and reach the point where they have to say something.
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  22. Agree seuss.
    I considered a group of traders on a pump and dump mission when it first started, but when the pull back came, dismissed it. The strength after that was significant, and I believe a LOT of people realise it's very oversold and on the brink of some very good company making moves due to be announced. Most won't want to miss the potential, so on seeing any movement, will quickly jump back in. That's no pump and dump.
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  23. I know. Maybe I didn't explain myself very well.
    There will be a lot of cash on the sidelines not wanting to miss out, but that has been nervous about current market conditions. Movement in stock price is enough to bring that money back in. Nothing to do with management, just investor psychology imo.
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  24. I believe you'll find that we now have SUPPORT at 10c.
    Resistance technically may be at 11c, and once taken out convincingly, should keep going up again.
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  25. Do you have a 2.7 million deposit for a new home?
    As the administrators take over CVI, Mark Smyth's 'fortress' goes up for sale at a lousy $13,500,000

    Now, with a 2.7million deposit, and interest rate of 7.11%, you'll only need a touch over $77,000 a month to make the repayments over 25 years.

    Feeling sick enough yet?
    Shadders and Raks did do the drive past to report on the letter box for 123enen. I remember it well from just after the EGM days.

    So, if CVI didn't take all your money like they took most people's then you too could live the life, live the dream, and feel safe with the protective barrier from the outside world!

    Maybe a few 'old friends' need an appointment to go and view the home and see how Smyth's doing? Is the dementia well advanced yet? Any house guests? Malcolm Johnson, Anton Tarkanyi, excelsior perhaps?

    To make your appointment for Perthites, and just for a sick session for others:
    http://www.domain.com.au/Property/For-Sale/House/WA/Mosman-Park/?adid=2008821829

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  26. tvp
    No answer from Arttse on that yet.......................
    Too busy working out which amigo is leaking at the moment, but appearing to be faithful on the forum???

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  27. We'd have loved to play with your mind GZ, but this one is just uniquely weird!

    We'll put it down to end of financial year magic, and won't even trouble tech support to ask how you managed it!

    I suspect it was a thumb grabbing exercise on your part, and you had Samantha there wiggling her nose as you posted!
    Hmmm. That's my best conspiracy theory for now!
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  28. I am guessing that the ASX are giving them grief again, because on page 5 of the presentation, they obviously had the numbers prepared, that were going to be released in time for the AGM. (Obviously again is my guess)

    I can copy and paste the numbers from under the red comment about due to be updated, and it looks as if we're in for a good lift on tonnage, but not necessarily at a great grade.
    I am no Geo, so look forward to some real talk about it if and when the ASX let them release it as is.

    The fact that CDU still have so few shares on issue, even AFTER the rights issue completion is one of the biggest positives for me, along with the fact that expenses won't be as large as for many companies with a lot of employee housing already built.

    Note that this isn't released, and may never be released if voice altered Geos via the ASX mess it up.
    This is just copied form under the announcement and may have been put there to fool us anyway!

    30.3mt @ 1.7% CuEq
    (0.8% cut-off) Measured and Indicated
    97.9mt @ 0.96% CuEq
    (0.4% cut-off) Measured and Indicated
    272.9mt @ 0.62% CuEq
    (0.2% cut-off) Measured & Indicated and inferred
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  29. I find that post rather repugnant and cynical cusox.
    Right now, imo it's a buy.

    What does that have to do with anything else?
    Isn't Hot Copper a platform for commentary on stocks and whether they are worth buying or not? If we didn't comment, there would be no Hot Copper

    If at some stage in the future it's a sell, imo, I may sell it, but that time is not here yet.
    Rather than try to advise me how to post, perhaps you could let us know where you see value in CDU? Do you wait for it to be proven and moving up again?

    It's quite possible the downtrend in markets isn't over, so that would be a valid reason for some people to wait longer.
    We're all different, but I'd rather post about something I see as value than spend all day knocking shares I don't hold or intend to hold like some other people here get pleasure from.

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  30. Shadow, that is bull dust, and you know it.
    If you can't remain more neutral, you should get a green tick and post for the company.
    You simply can't give a value on it without ALL the information.
    Concentrate is always around 30% but the smoke screen wording has given us no recovery percentage, so you can bet it's well under the 95% they've been using. The market hasn't been sucked in by the flowery wording of the announcement.
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  31. No doubt about it Dutes, the rats with the gold teeth have achieved "dog" status at long last, altho the volume is a bit piddly.

    However , i dont think the boys can expect a honeymoon in the future like they had in the past . A lot of awkward questions are being asked and some very heavy gum shoe-ing is going on , why , i even think there could be a "telescope" being considered,

    Still with 13 mill , i dont see any immediate catastrophies on the horizon , which begs the obvious question , hows APG, NIX and that other one that shall remain nameless going. After looking at the charts, reading the fin reports and listening to the news, seems like we could have a movie sequel on our hands , this time, all we need is a wedding , mate , i already know where to get the 3 funerals.

    Cheers

    OI NQ , how they hanging?

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  32. Announcement from ERM has made my day. :)

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  33. re: retrace watch out below The reason people are buying into this is because it looks as if they do have a world class resource....if that is the case this stock is very undervalued at current levels.
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  34. tvp
    Maybe this sheds some light on it ............................
    He was suspected of being Bendigo. Maybe the mods worked it out.

    Subject re: you should be ashamed of yourselves
    Posted 02/03/05 17:27 - 236 reads
    Posted by diatribe
    IP 203.51.xxx.xxx
    Post #529197 - in reply to msg. #529196 - splitview

    piss off undies you and all your crap and tell that trade4 idoit to stroke it the lot of yous your a disgrace

    Voluntary Disclosure: No Position Sentiment: None TOU violation






    Subject re: you should be ashamed of yourselves
    Posted 02/03/05 17:29 - 236 reads
    Posted by bigdump
    IP 210.49.xxx.xxx
    Post #529199 - in reply to msg. #529188 - splitview

    so who should be ashamed of themselves
    it squite ironic !
    Isn't talking to ones self a form of madness





    Voluntary Disclosure: No Position Sentiment: None TOU violation






    Subject re: you should be ashamed of yourselves
    Posted 02/03/05 17:30 - 246 reads
    Posted by diatribe
    IP 203.51.xxx.xxx
    Post #529201 - in reply to msg. #529199 - splitview

    fark u 2 fool ramper

    Voluntary Disclosure: No Position Sentiment: None TOU violation






    Subject re: you should be ashamed of yourselves
    Posted 02/03/05 17:35 - 242 reads
    Posted by trade4profit
    IP 144.139.xxx.xxx
    Post #529204 - in reply to msg. #529197 - splitview

    diatribe...

    Here are the posts you refer to "6 - 8 weeks ago"...

    ---

    Subject copper strike.. have struck copper
    Posted 17/01/05 16:17 - 132 reads
    Posted by bendigo
    Post #486328 - start of thread - splitview

    Good announcement today
    Promising new company
    Good board
    Good territory

    go the ASX website & check out the announcment.

    Cheers
    Bendigo

    ---

    Subject re: copper strike.. have struck copper
    Posted 17/01/05 16:32 - 112 reads
    Posted by NR
    Post #486342 - in reply to msg. #486328 - splitview

    all ready on them bendigo......awaiting further annonucements.......


    ---


    Subject re: copper strike.. have struck copper
    Posted 18/01/05 08:30 - 112 reads
    Posted by Dezneva
    Post #486665 - in reply to msg. #486328 - splitview

    Yep, I agree. I know the people as well. They have a whole heap of old TEC ground. Its a great hit. and I think they are continuing the drilling.

    ---


    These were the first 3 posts ever on CSE.

    Although Dezneva only posted "...I know the people as well...", I can see how you may have remebered that as "...the boss being a good bloke..."

    Problem is, it was Bendigo he was replying to and not you!

    How do you explain that?

    Cheers!

    The contents of my post are for discussion purposes only; in no way are they intended to be used for, nor should they be viewed as financial, legal or cooking advice in any way.

    Voluntary Disclosure: No Position Sentiment: None TOU violation






    Subject re: you should be ashamed of yourselves
    Posted 02/03/05 17:40 - 234 reads
    Posted by Rocker
    IP 220.253.xxx.xxx
    Post #529215 - in reply to msg. #529204 - splitview

    well picked up T4P


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  35. I get your drift joewolf.
    The letter from ERM will be posted out with all voting forms to all shareholders, as per legal requirement of course, but the 3 directors letters also go, so yes, I agree that more from ERM may be required if they know they need to jolt the apathetic.

    Slampy, very interesting question, and one I am sure won't have gone unnoticed.

    Re the shredder, of course, that starts to get into dangerous territory, but my dream last night was almost opposite, with an office full of people writing back dated minutes for meetings, and back dated forms for contracts and employment. It was a hectic dream, and I hope there's no reality in it at all.


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  36. I reckon you should all get a life personally!
    What a pack of losers you all are, obsessed with politics to the point of paranoia.
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  37. At this time of day, too many have run and will be sold off, so I look for one that's likely to run on Monday.

    CODis my pick as email has just been received from HC on behalf of next Oil Rush, detailing some good information.

    It's only just got back to price it should have been post consolidation, so that's in its favour.
    Very little to sell, I like that, as it will move quickly.

    Many won't have received the email yet as they're at work, etc.

    Read more here.

    http://www.nextoilrush.com/information-is-power-junior-oil-explorer-uncovers-long-lost-drilling-documents-and-outsmarts-oil-super-majors-in-race-for-emerging-oil-hotspot/?utm_source=HCMO

    Looks good for next week. Be prepared!
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  38. 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 19 20
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  39. tvp
    re: it goes like this? Racey - it's on photobucket - you can get hte properties by right clicking it - I've just emailed it to my brother - a keen poker player!

    Salty - howsabout an email update please imo!!
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  40. 6,293 Posts.
    lightbulb Created with Sketch. 22316
    I thought it could be worthwhile to provide some information on another promising Fragile-x drug which ultimately didn’t succeed in making it to market.

    Seaside Therapeutics is a privately owned US-based biotech. Seaside was initially developing two separate compounds for the treatment of Fragile-x and autism. The first was STX209 (arbaclofen), a selective agonist of **A-B. The second was STX107, a selective mGluR5 antagonist.

    In pre-clinical testing, STX209 was shown to reverse both neuropathological and behavioral abnormalities in an Fmr1-knockout mouse model. Based on this positive data, clinical trials of STX209 were initiated.

    By 2010 Seaside had announced results from a Phase 2 trial of STX209 in Fragile-x. The trial included 63 adults and children (6-39 years old) in a multi-site, randomised, double blind, placebo- controlled, crossover study conducted in the US over 15 months. Dosing was flexible, with tapering up of the drug every 3–4 days to a maximum of 10 mg twice a day for children and 10 mg three times per day for adults until the optimal tolerated dose was established. The participants were evaluated at 2 and 4 weeks after starting the treatment and then tapered off the treatment over 1–2 weeks. There was a 7-day washout period between the placebo and active treatments. Assessment measures included the Clinical Global Impression Scale (CGIS), the Aberrant Behavior Checklist (ABC), Vineland Adaptive Behavior Scales (Second Edition; VABS), the Social Responsiveness Scale (SRS), the Repetitive Behavior Scale-Revised, the Child and Adolescent Symptom Inventory Anxiety Scale, the Attention Deficit Hyperactivity Disorder Rating Scale-IV, a measure of vocabulary and short-term and working memory, and a Visual Analog Scale (VAS) of the child’s three most problematic behaviors.

    The trial failed to meet its primary endpoint, which was the irritability subscale of the ABC. However, on one secondary endpoint, the VAS problem behaviour rating, results were significantly better with the active treatment than with placebo. Another secondary measure, the social avoidance ABC subscale, was also found to be significantly improved during active treatment as compared to placebo. In addition, separate analysis of participants with the worst social impairments showed that they displayed significant improvement during active treatment as compared to placebo.

    The research was presented at an International Fragile-x conference by by Elizabeth Berry-Kravis and Randi Hagerman, both of whom are quoted in Neuren’s announcement this week. They described the trial results and parental reports of improvement as “exciting”.

    A few months later, Seaside announced positive data from an open-label Phase 2 study of STX209 conducted in patients with autism spectrum disorders (ASD). This time, the primary endpoint of the study, an improvement on the Irritability subscale of the Aberrant Behavior Checklist (ABC-I), achieved statistical significance (p<0.001). In addition, STX209 demonstrated statistically significant improvements across a number of other global and specific neurobehavioral outcomes, including improvements on the Social Withdrawal subscale of the Aberrant Behavior Checklist (ABC-SW, p<0.001), which assesses a core symptom of ASD.

    In 2012, Seaside announced that it had struck a deal with Roche. Financial terms were not disclosed. Under the terms of the deal, Seaside agreed to license its STX107 (mGluR5) compound to Roche and Roche took over responsibility for the development and commercialisation of STX107 for Fragile-x and ASD. At this time, Roche was already developing its own mGluR5 drug candidate, RG7090, for Fragile-x. Under the agreement with Roche, Seaside was to complete development of STX209 and Roche took an option to take the drug to market, if successful.

    As results of the Phase 2 studies of STX209 in both Fragile-x and ASD were considered to be highly promising, Seaside had commenced a Phase 3 trial of STX209 in Fragile-x and a further Phase 2b trial in ASD.

    But the studies were suddenly terminated in 2013. Seaside said that termination wasn’t due to safety issues. At first the company seemed to blame the termination on financial reasons. Demonstrated efficacy then became the issue. According to the VP of Seaside

    The FDA requires companies to pick one, and only one, assessment as the “primary endpoint” of the study. In their eyes, the result on that one pre-selected endpoint makes or breaks the study. In our recent autism study, STX209 did not show an advantage over placebo on the primary endpoint of social withdrawal, so the FDA and some news reporters regard it as a negative study. In fact, STX209 did show advantages over placebo on a number of other assessments. Some of these secondary endpoints are just as meaningful as the social withdrawal assessment, or even more so, but in the FDA’s eyes, they don’t make the study a positive study, because they were not pre-selected as the “primary endpoint.

    Parents of children who had participated in the trials were devastated. Many saw significant improvement in their children during the trials. They wrote an open letter to Roche, pleading for funding to continue the development of arbaclofen, with no success. They also told their stories on a dedicated website. The following are some examples.

    Within the first 2 weeks we had a birthday party for his younger brother and our annual Super Bowl party (both involve guests and noise) he did not however retreat to his room, he stayed with us, he participated. Two weeks after that I sat in my vehicle and cried as I waited for him to walk to the car after school. For the first time ever, he walked out of the school with two other boys (classmates) and they were talking and laughing with one another. It was later that same week that he told my husband that the kids at school were not making fun of him anymore


    Now that the medicine is out of her system, MY L is gone. It’s only been 2 weeks since she titrated down. It’s only been 30 hours since she took her last pill. I’ve already seen more meltdowns, more fits, more “I can’ts”, more hitting, and more biting attempts than I saw in the last year combined.



    He was like a different child. He enjoyed going to school and being with people. His communication increased and his anxiety descreased. We could run errands. Waiting in lines and getting past automatic doors became doable. The IEP vision statement we came up with started to look like a real possibility. He already had the friend part down. And he is a valued member of his class.



    Then we received the devastating news that the STX209 trial was discontinued. Blessedly, we were able to get through the school year before having to start weaning him. But once we did, we started seeing those old behaviors and challenges returning



    I waited 10 and a half years for him to tell me he loved me…. With fragile X, you’re like living in a box and someone is holding the lid down. The medication opened the lid and let Parker out….I don’t want to go back to the way life was.

    Dr. Mike Tranfaglia, whose son has fragile X but wasn’t in the Seaside trial, said that STX209 appeared to significantly help about a third of patients but also made some patients worse. He said that without being able to tell in advance which patients would benefit, it would be hard for the drug to succeed in a clinical trial and win approval.

    Around the same time, Novartis was releasing results of its Phase IIb/III studies with mavoglurant (AFQ056) - an mGluR5 therapy- in separate studies in adolescents and adults with Fragile- x . Both studies failed to meet the primary endpoint of showing significant improvement in abnormal behaviours compared to placebo. Novartis terminated the program.

    Late last year, Roche added to the string of disappointing results when it announced that both of its Phase 2 studies for its mGluR5 therapy - RG7090 -had failed to meet both primary and secondary endpoints. It also shut down its Fragile-x program.

    Like TBI, clinical development of Fragile-x therapies has proven to be incredibly challenging. To date, no one has successfully completed a Phase 3 trial. This is both Neuren’s opportunity and its challenge. In the context of the recent history of Fragile-x clinical trials, it is inevitable that many will approach this week’s Phase 2a results with cautious optimism. Trofinetide’s success in Phase 2 trials for conditions which represent both ends of the autistic spectrum is certainly encouraging, as are the improvements reported by parents of participants.

    But, as we were reminded in this week's announcement, it is only the first step.


    http://www.seasidetherapeutics.com/

    http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4025936/

    http://blog.autismspeaks.org/2010/07/

    http://www.roche.com/media/store/releases/med-cor-2012-06-19.htm

    http://www.nytimes.com/2013/06/07/business/an-experimental-drugs-bitter-end.html?hp&_r=0

    http://www.ageofautism.com/2013/06/...rug-trial-for-fragile-x-parents-dismayed.html

    http://stx209stories.com/stx209/rhett-murphy-holton-ks/

    http://www.fiercebiotech.com/story/...rogram-after-phii-trials-flunk-out/2014-09-10

    https://fragilex.org/2014/research/...-conclusion-of-the-long-term-extension-study/
  41. What a fascinating thread reading back 3 months!

    Lots of reading today!
    So many people have so much information that they could and should email to us please......

    [email protected]

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