|By PR Newswire||
|February 10, 2014 05:27 PM EST||
WASHINGTON, Feb. 10, 2014 /PRNewswire-iReach/ -- Health staffers from over 80 Congressional offices together with agency and industry officials jammed a briefing room last Friday to hear mothers, clinicians and leading researchers call on the FDA to accept and promptly review a new drug application for eteplirsen, a promising new treatment for Duchenne Muscular Dystrophy that could save thousands of lives. Sponsored by Rep. William Keating (D-MA-9th), and Rep. Spencer Bachus (R-AL-6), the briefing featured three of seven leading scientists who briefed the FDA the previous day, and previewed an upcoming documentary on the lives of the children who suffer from Duchenne.
Duchenne is the leading genetic killer of children, affecting about one out of every 3,500 boys born in the United States. With no approved treatment, Duchenne has a 100 percent fatality rate. A child of five faced with Duchenne experiences a loss of muscle strength that leads to confinement to a wheelchair by adolescence and a shortened life assisted by ventilators. The progressive deterioration of muscle strength leads to death in the late teens or early twenties.
"Our children are facing the most common fatal childhood disorder in the world," said Christine McSherry. "There is no cure for my son Jett, but there is a therapy that could save his life."
In 2012 Congress granted the FDA special authority to accelerate approval of treatments that demonstrate real promise in mid-phase trials for diseases like DMD that are rare and fatal. Pointing to the FDA's power to accelerate therapy approval, Mindy Leffler whose son Aidan suffers from Duchenne introduced the expert panel representing decades in Duchenne research and clinical trials. Noted Leffler, "The drug's effect in the 12 boys in the trial is obvious and extraordinary. We were concerned that the FDA might be uncomfortable listening to us as parents, so we brought the world's leading experts in the treatment and research of Duchenne to them."
Dr. Jerry Mendell, the principal investigator for the breakthrough drug eteplirsen, with the most clinical trials through three decades of clinical research declared, "This drug works."
Also briefing was Harvard Medical School Professor, Dr. Louis Kunkel, the leading authority who discovered in 1986 that Duchenne was caused by a "dystrophin" deficiency. Kunkel described the boys receiving the new therapy by saying, "They are stabilized. They are making dystrophin. This is really quite an amazing feat." "I support this," he stated.
Australian Professor Steve Wilton, a neuromuscular NIH funded researcher and one of the world's pioneers of "exon skipping" gene therapy, described eteplirsen's promise, "In Australia we'd say it's bleeding obvious."
Summarizing the case before the FDA, Dr. Mendell said, "Clearly it's a breakthrough drug, it has minimal side-effects. I say minimal, we haven't seen a single one, which is incredibly remarkable, and we unequivocally showed efficacy over a two year period which is unheard of in clinical trials. If you add up all these things, that is the ticket for accelerated approval by the FDA, and we need help getting there."
The scientists saw a decisive decade for Duchenne research and the parents saw a pivotal moment, where a FDA decision could determine whether their children are part of the last generation to die from Duchenne, or among the first generation to live.
Tracy Seckler, mother of 13 year-old Charley with Duchenne, summarized by saying, "You've just heard from the world's experts who are are fulfilling their responsibility as outspoken advocates for kids with Duchenne. They traveled here from Boston, Columbus and Perth because in all their collective 95 years of experience, they have never seen clinical trial results like this." In closing, Tracy realized the implications for Charley and the other children, "We face two timelines to FDA approval, one accelerated, which will give children this drug by end of this year, or the business as usual timeline, with full phase three trials, meaning no access to this drug until 2018. That's four years that children like Jett do not have."
Jenn McNary, with two sons, Austin 15 and Max 12, one of whom has benefited from the eteplirsen trial noted, "We live with the science every day in our home. We are not so much in a race against the science as we are in a race against the clock. Will the FDA approve this in time for the families sitting in this room?"
For an edited video of the briefing go to: http://youtu.be/DTZizFZOtQY
To view this video on YouTube, please visit: http://youtu.be/DTZizFZOtQY
Media Contact: Norris Clark, Princeton Strategic Communications, 609-425-1325, [email protected]
News distributed by PR Newswire iReach: https://ireach.prnewswire.com
SOURCE Princeton Strategic Communications
SYS-CON Events announced today that robomq.io will exhibit at SYS-CON's @ThingsExpo, which will take place on June 9-11, 2015, at the Javits Center in New York City, NY. robomq.io is an interoperable and composable platform that connects any device to any application. It helps systems integrators and the solution providers build new and innovative products and service for industries requiring monitoring or intelligence from devices and sensors.
Apr. 1, 2015 06:00 PM EDT Reads: 1,610
The 17th International Cloud Expo has announced that its Call for Papers is open. 17th International Cloud Expo, to be held November 3-5, 2015, at the Santa Clara Convention Center in Santa Clara, CA, brings together Cloud Computing, APM, APIs, Microservices, Security, Big Data, Internet of Things, DevOps and WebRTC to one location. With cloud computing driving a higher percentage of enterprise IT budgets every year, it becomes increasingly important to plant your flag in this fast-expanding bu...
Apr. 1, 2015 05:15 PM EDT Reads: 553
Today, IT is not just a cost center. IT is an enabler and driver of business. With the emergence of the hybrid cloud paradigm, IT now has increasingly more capabilities to create new strategic opportunities for a business. Hybrid cloud allows an organization to utilize multi-tenant public clouds, dedicated private clouds, bare metal hosting, and the associated support and services for the right use cases through an on-demand, XaaS model. This model of IT creates tremendous opportunities for busi...
Apr. 1, 2015 05:00 PM EDT Reads: 3,475
Business as usual for IT is evolving into a “Make or Buy” decision on a service-by-service conversation with input from the LOBs. How does your organization move forward with cloud? In his general session at 16th Cloud Expo, Paul Maravei, Regional Sales Manager, Hybrid Cloud and Managed Services at Cisco, discusses how Cisco and its partners offer a market-leading portfolio and ecosystem of cloud infrastructure and application services that allow you to uniquely and securely combine cloud busi...
Apr. 1, 2015 04:15 PM EDT Reads: 1,658
SYS-CON Events announced today that Litmus Automation will exhibit at SYS-CON's 16th International Cloud Expo®, which will take place on June 9-11, 2015, at the Javits Center in New York City, NY. Litmus Automation’s vision is to provide a solution for companies that are in a rush to embrace the disruptive Internet of Things technology and leverage it for real business challenges. Litmus Automation simplifies the complexity of connected devices applications with Loop, a secure and scalable clou...
Apr. 1, 2015 04:00 PM EDT Reads: 789
Businesses are looking to empower employees and departments to do more, go faster, and streamline their processes. For all workers – but mobile workers especially – utilizing the cloud to reconnect documents and improve processes without destructing existing workflows can have a dramatic impact on productivity. In his session at 16th Cloud Expo, Mark Grilli, vice president of Acrobat Solutions marketing at Adobe Systems Incorporated, will outline new ways that the cloud is changing the way peo...
Apr. 1, 2015 04:00 PM EDT Reads: 1,452
With the arrival of the Big Data revolution, a data professional is expected to master a broad spectrum of complex domains including data processing, mathematics, programming languages, machine learning techniques, and business knowledge. While this mastery is undoubtedly important, this narrow focus on tool usage has divorced many from the imagination required to solve real-world problems. As the demand for analysis increases, the data science community must transform from tool experts to "data...
Apr. 1, 2015 03:45 PM EDT Reads: 832
As Marc Andreessen says software is eating the world. Everything is rapidly moving toward being software-defined – from our phones and cars through our washing machines to the datacenter. However, there are larger challenges when implementing software defined on a larger scale - when building software defined infrastructure. In his session at 16th Cloud Expo, Boyan Ivanov, CEO of StorPool, will provide some practical insights on what, how and why when implementing "software-defined" in the dat...
Apr. 1, 2015 03:45 PM EDT Reads: 578
While not quite mainstream yet, WebRTC is starting to gain ground with Carriers, Enterprises and Independent Software Vendors (ISV’s) alike. WebRTC makes it easy for developers to add audio and video communications into their applications by using Web browsers as their platform. But like any market, every customer engagement has unique requirements, as well as constraints. And of course, one size does not fit all. In her session at WebRTC Summit, Dr. Natasha Tamaskar, Vice President, Head of C...
Apr. 1, 2015 03:45 PM EDT Reads: 685
SYS-CON Events announced today that Emcien will exhibit at SYS-CON's 16th International Cloud Expo®, which will take place on June 9-11, 2015, at the Javits Center in New York City, NY. Emcien’s vision is to let anyone use data to know the future. Emcien has built an automated, predictive analysis product that improves the lives of real people. Emcien allows people to automate their data analysis so they can build a better future.
Apr. 1, 2015 03:00 PM EDT Reads: 858
SYS-CON Events announced today that Solgenia will exhibit at SYS-CON's 16th International Cloud Expo®, which will take place on June 9-11, 2015, at the Javits Center in New York City, NY, and the 17th International Cloud Expo®, which will take place on November 3–5, 2015, at the Santa Clara Convention Center in Santa Clara, CA. Solgenia is the global market leader in Cloud Collaboration and Cloud Infrastructure software solutions. Designed to “Bridge the Gap” between Personal and Professional S...
Apr. 1, 2015 03:00 PM EDT Reads: 3,104
WSM International has launched a DevOps services division that offers assessment, consulting and implementation to large enterprises and organizations with complex infrastructures. The concept of DevOps is to blend information technology (IT) software development with operations to optimize the computing infrastructure according to the specific needs of the organization. According to a recent press release from Gartner, "By 2016, DevOps will evolve from a niche strategy employed by large cloud ...
Apr. 1, 2015 03:00 PM EDT Reads: 1,477
SYS-CON Events announced today that QTS Realty Trust, one of the nation’s largest and fastest-growing providers of data center facilities and cloud services and a leader in security and compliance, will exhibit at SYS-CON's 16th International Cloud Expo®, which will take place on June 9-11, 2015, at the Javits Center in New York City, NY. QTS Realty Trust, Inc. (NYSE: QTS) is a leading national provider of data center solutions and fully managed services, and a leader in security and compliance...
Apr. 1, 2015 03:00 PM EDT Reads: 1,421
SYS-CON Events announced today that WSM International (WSM), the world’s leading cloud and server migration services provider, will exhibit at SYS-CON's 16th International Cloud Expo®, which will take place on June 9-11, 2015, at the Javits Center in New York City, NY. WSM is a solutions integrator with a core focus on cloud and server migration, transformation and DevOps services.
Apr. 1, 2015 03:00 PM EDT Reads: 1,459
SYS-CON Events announced today that MangoApps will exhibit at SYS-CON's 16th International Cloud Expo®, which will take place on June 9-11, 2015, at the Javits Center in New York City, NY., and the 17th International Cloud Expo®, which will take place on November 3–5, 2015, at the Santa Clara Convention Center in Santa Clara, CA. MangoApps provides private all-in-one social intranets allowing workers to securely collaborate from anywhere in the world and from any device. Social, mobile, and eas...
Apr. 1, 2015 03:00 PM EDT Reads: 3,277