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PTC Therapeutics, Inc. (PTCT)

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17.50-0.06 (-0.34%)
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  • M
    M
    M
    Great results
  • H
    Harry
    Harry
    A memo written by John Jenkins at the FDA has come to light casting doubt that Translarna can be approved. It basically lambasts the way Sarepta was approved despite obviously not been effective. Translarna has failed trials many times but PTC and advocates won't give up. The FDA probably is reviewing it in September so they can put this thing to bed once and for all.
  • K
    KindI
    KindI
    Dr. Tuyen Ong resigned as PTCT's chief medical officer, right before the FDA meeting. It doesn't take a genius to figure out that PTCT is totally f**ked up!! Come on ...
  • C
    Chung
    Chung
    There is not a single study that shows Translarna works. It is an "expensive placebo"!!
  • E
    Equity Daily
    Equity Daily

    [!] Option spread traders take a look at CreditSpreads.io, zero cost live table of statistically profitable option credit spreads for the next 4 weeks, sometimes including spreads for PTCT! High probability option credit spreads deliver a solution to invest with defined risk and the maximum profit gained up front. CreditSpreads.io lazer-targets exclusively the option spreads which meet its rigid standards for probability and profit, and ensures sustained profitability by alerting you of any impending events which could affect the trade. http://www.creditspreads.io

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  • K
    KindI
    KindI

    Here is the news:
    http://www.businesswire.com/news/home/20170801006061/en/Nightstar-Appoints-Tuyen-Ong-Chief-Development-Officer

    Nightstar Appoints Tuyen Ong as Chief Development Officer
    Nightstar Appoints Tuyen Ong as Chief Development Officer
    www.businesswire.com
  • B
    BOOSKIADAM
    BOOSKIADAM
    He was hired by another company. Not much PTC can do about that......... Sky is NOT falling.
  • O
    Olivia
    Olivia
    The upside potential for PTCT just got a whole lot more interesting. if you guys have questions you should ask Awe.Some.Stock.s. They often respond to my emails which is helpful.
  • M
    Maria
    Maria
    Perhaps a short-term pull back is more likely before the next rally on PTCT? Yo you should really check out awe*some*stock*s, they seem on point with their stocks.
  • A
    AndrewJ5267
    AndrewJ5267

    I'm just listing the Ataluren trial results (http://www.thelancet.com/journals/lancet/article/PIIS0140-6736%2817%2931611-2/abstract):
    −7·7 m (SE 24·1, 95% CI −54·9 to 39·5; p=0·749) in the group with a 6MWD of less than 300 m,
    42·9 m (15·9, 11·8–74·0; p=0·007) in the group with a 6MWD of 300 m or more to less than 400 m, and
    −9·5 m (17·2, −43·2 to 24·2; p=0·580) in the group with a 6MWD of 400 m or more

    Change in 6MWD did not differ significantly between patients in the ataluren group and those in the placebo group, neither in the intention-to-treat population nor in the prespecified subgroups with a baseline 6MWD of
    - less than 300 m {−7·7 m}, or
    - 400 m {−9·5 m} or more.
    However, we recorded a significant effect of ataluren in the prespecified subgroup of patients with a baseline 6MWD of
    - 300 m or more to less than 400 m {42·9 m}

    Looking at the results, yes, I'd have to agree that the '300 m or more to less than 400 m' group demonstrated a significant, positive result from the treatment. By all means, I believe children in this group should be given access to this treatment.

    Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
    Change in 6MWD did not differ significantly between patients in the ataluren group and those in the placebo group, neither in the intention-to-treat population nor in the prespecified subgroups with a baseline 6MWD of less than 300 m or 400 m or more
    www.thelancet.com
  • B
    BOOSKIADAM
    BOOSKIADAM
    Must be riding the coat tails of SRPT.
  • J
    Johnnie Ringo
    Johnnie Ringo
    Wow. PTCT got a favorable FDA review for Translarna. Somehow everybody else knew this and I missed all announcements and headlines completely. More how the crooked market operates. The retail investor is always the last to find out. Still good news!
  • K
    Kathy D
    Kathy D
    Down on no news!
  • B
    BOOSKIADAM
    BOOSKIADAM
    BEAR RAID before BLAST OFF !
  • A
    Anonymous
    Anonymous

    Option traders pay a visit to CreditSpreads.io, zero cost live feed of mathematically gainful option credit spreads for the following four weeks, sometimes including spreads for PTCT! High probability option credit spreads offer you an effective way to invest with specified risk and the maximum profit received up front. CreditSpreads.io targets solely the credit spreads which meet its strict requirements for risk and profit, and contributes to sustained profitability by telling you of any impending catalysts that may affect the trade. http://creditspreads.io

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  • K
    Kathy D
    Kathy D
    whats going on?
  • A
    AndrewJ5267
    AndrewJ5267
    Preliminary Phase 2 Data from Spinal Muscular Atrophy Program Presented at CureSMA Conference

    10:20 am ET July 1, 2017 (PR Newswire) Print
    PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the presentation of preliminary clinical data from the company's joint development program with Roche and the SMA Foundation in spinal muscular atrophy (SMA) at the Cure SMA Meeting in Orlando, FL. Preliminary results from an early analysis of Part 1 of the Phase 2 SUNFISH trial evaluating oral RG7916, a small molecule modifier of Survival Mo tor Neuron 2 (SMN2) splicing, were highlighted in an oral presentation titled "Clinical Studies of RG7916 in Patients with Spinal Muscular Atrophy: Study Update." SMA is a rare genetic disorder that results in neuromuscular disability beginning in infancy and is the leading genetic cause of mortality in infants and young children.

    "The increase in SMN2 full length transcript in SMA patients is promising and confirms that RG7916 targets the underlying cause of the disease," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "The low levels of SMN protein in SMA patients affect multiple tissues throughout the body, including muscles, bones and nerves. We believe that the ease of administration of an oral therapy and its broad tissue distribution give RG7916 the potential to address the complete spectrum of functional deficits observed in SMA patients. We look forward to advancing RG7916 into pivotal clinical trials in the second half of the year."

    Preliminary results from an early analysis from the ongoing Part 1 of the RG7916 SUNFISH study in Type 2/3 SMA patients demonstrated a dose-dependent increase in SMN2 full length/7 mRNA ratio of ~ 400% versus baseline, as measured in whole blood. These results provided proof of mechanism for oral small molecule SMN2 splicing modifiers. No drug-related adverse events leading to withdrawal have been observed to date for RG7916.

    SUNFISH is a two-part phase 2 clinical study. Part 1 is a double-blind, placebo-controlled, randomized, exploratory dose-finding study in Type 2/3 pediatric and adult SMA patients. The primary objective of the first part of the study is to evaluate the safety, pharmacokinetics, and pharmacodynamics of RG7916 in patients, and to select the dose for the second part of the study. The pivotal second part is a double-blind, placebo-controlled, randomized, confirmatory study in Type 2/3 SMA patients followed by an open-label extension. The primary objective of the pivotal second part of the study is to evaluate the safety and efficacy of RG7916 compared to placebo. SUNFISH is one of three ongoing clinical trials of RG7916, along with FIREFISH and JEWELFISH.

    The SMA program was initially developed by PTC Therapeutics in partnership with the SMA Foundation in 2006 to accelerate the development of a treatment for SMA. In November 2011, Roche gained an exclusive worldwide license to the PTC/SMA Foundation SMN2 alternative splicing program. The development of these compounds is being executed by Roche and overseen by a joint steering committee with members from PTC, Roche, and the SMA Foundation.
  • J
    JBS Inc.
    JBS Inc.
    PTCT been running good for while ; )
  • Y
    Yahoo Finance Insights
    Yahoo Finance Insights
    PTCT reached a new 52 Week High at 18.63
  • K
    Kathy D
    Kathy D
    Up and away!