20th International WMS Congress, Wednesday 30th September- Sunday 4th October, 2015 Brighton, UK
A 4-Day Symposium of the World Muscle Society in association with its official journal Neuromuscular Disorders
The symposium will be in the traditional WMS format with 3 selected topics:
The Congress will be held in the traditional WMS format with three selected topics. One day of the symposium will be dedicated to each of the selected topics addressing emerging discoveries in the field:
Muscle metabolism and exercise in health and disease
Immune mediated Peripheral Nerve, Neuromuscular Junction, and Muscle Disorders
Advances in the treatment of Neuromuscular Disorders
Abstracts will also be welcome on advances across the neuromuscular field.
Victor Dubowitz (London), Peter van Den Bergh (Brussels), Thomas Voit (Paris), Haluk Topaloglu (Ankara), Francesco Muntoni (London)
LOCAL ORGANIZING COMMITTEE:
Francesco Muntoni (Chair) (UCL Institute of Child Health, London)
Ros Quinlivan (Vice Chair) (UCL Institute of Neurology, London)
Mike Hanna (UCL Institute of Neurology, London)
Mary Reilly (UCL Institute of Neurology, London)
Susan Brown (Royal Veterinary College London)
Heinz Jungbluth (Kings College London)
Gita Ramdharry (UCL Institute of Neurology, Kingston University and St George's, University of London)
Caroline Sewry (UCL Institute of Child Health)
Opening of Registration and Abstract Submission: 1 December 2014
Abstract Deadline Submission: 2 April 2015
Early Bird Registration Deadline: 30 April 2015
Regular registration opens: 1 May 2015
Pre-Congress Course: 29-30 September 2015
Sarepta's stock could soar with a regulatory approval for its experimental DMD drug
Sarepta's shares have already climbed by a staggering 139% this year, after the company announced that the FDA agreed to review its exon-skipping Duchenne muscular dystrophy, or DMD, drug, eteplirsen. Shorts, though, still hold a massive position in the stock, with over 35% of the float being sold short. As a reminder, eteplirsen's regulatory pathway has been hindered by the fact that Sarepta has only completed a midstage study that included a grand total of 12 patients.
While there's a good chance that the FDA could still decide to reject the application outright or issue a Complete Response Letter following a formal review, the obvious risk for short-sellers is that eteplirsen's limited data turn out to be enough to convince the agency that its benefits outweigh the risks for a disease that is always fatal. Perhaps the biggest issue that should be keeping short-sellers up at night is that an eteplirsen approval may validate Sarepta's remaining DMD pipeline that seeks to treat upwards of 80% of this patient population -- meaning that its DMD platform has the potential to rake in billions in sales.
More than revenues, losses or cash reserves, that was the piece of news that caught investors’ attention this morning when Sarepta Therapeutics (Nasdaq: SRPT) gave its quarterly update. The trial on which the company will rely heavily for approval of its drug to slow the progression of Duchenne muscular dystrophy has been ongoing for almost four years now. The 10 boys taking part in that part of the trial (two were excluded years ago from the averages because they lost their ability to walk early on) have been tested every six months to see how far they can walk over the course of six minutes to measure how fast their muscles are deteriorating due to the disease.
While the company did not disclose the exact results of that trial of 10 boys, the fact that all are still able to walk at all is good news. The average age of the 10 boys is now more than 12 years, the average age at which boys with Duchenne become wheelchair-bound due to the disease. That fact is likely the cause of the 2 percent share increase today as of noon at $33.38, within a few cents of the company’s highest share price in the past year.
Interim CEO Ed Kaye disclosed the fact on a conference call with analysts this morning, and said that the company intends to make specifics of the trial results after 192 weeks (about three years and eight months) known before an FDA advisory committee meets to recommend whether the drug, called eteplirsen, should be approved, likely in late November. The agency has not given a specific date for the meeting, nor has it officially blessed Sarepta’s application for approval of the drug, but assuming it does, the full agency could approve the drug as soon as late February.
Different set of investors and SRPT is a bigger threat for all hedge funds that got accustomed to play it up and down.
Peter Fitzhugh Brown - Margie's husband. He is CEO of Renaissance Technologies hedge fund, which trades in markets around the world, and has employed complex mathematical models to analyze and execute trades, many of them automated. Renaissance uses computer-based models to predict price changes in easily traded financial instruments. These models are based on analyzing as much data as can be gathered, then looking for non-random movements to make predictions.[ Some also attribute Renaissance performance to employing financial signal processing techniques such as pattern recognition.
Presumably, SRPT was shorted by Brown with the tips from Margie. Crime yet to be punished.