|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
Fifty billion connected devices and still no winning protocols standards. HTTP, WebSockets, MQTT, and CoAP seem to be leading in the IoT protocol race at the moment but many more protocols are getting introduced on a regular basis. Each protocol has its pros and cons depending on the nature of the communications. Does there really need to be only one protocol to rule them all? Of course not. In his session at @ThingsExpo, Chris Matthieu, co-founder and CTO of Octoblu, walk you through how Oct...
Sep. 26, 2016 06:30 PM EDT Reads: 2,115
SYS-CON Events announced today that Secure Channels will exhibit at the 19th International Cloud Expo, which will take place on November 1–3, 2016, at the Santa Clara Convention Center in Santa Clara, CA. The bedrock of Secure Channels Technology is a uniquely modified and enhanced process based on superencipherment. Superencipherment is the process of encrypting an already encrypted message one or more times, either using the same or a different algorithm.
Sep. 26, 2016 05:15 PM EDT Reads: 1,581
Vidyo, Inc., has joined the Alliance for Open Media. The Alliance for Open Media is a non-profit organization working to define and develop media technologies that address the need for an open standard for video compression and delivery over the web. As a member of the Alliance, Vidyo will collaborate with industry leaders in pursuit of an open and royalty-free AOMedia Video codec, AV1. Vidyo’s contributions to the organization will bring to bear its long history of expertise in codec technolo...
Sep. 26, 2016 05:15 PM EDT Reads: 2,578
Major trends and emerging technologies – from virtual reality and IoT, to Big Data and algorithms – are helping organizations innovate in the digital era. However, to create real business value, IT must think beyond the ‘what’ of digital transformation to the ‘how’ to harness emerging trends, innovation and disruption. Architecture is the key that underpins and ties all these efforts together. In the digital age, it’s important to invest in architecture, extend the enterprise footprint to the cl...
Sep. 26, 2016 05:15 PM EDT Reads: 262
Traditional on-premises data centers have long been the domain of modern data platforms like Apache Hadoop, meaning companies who build their business on public cloud were challenged to run Big Data processing and analytics at scale. But recent advancements in Hadoop performance, security, and most importantly cloud-native integrations, are giving organizations the ability to truly gain value from all their data. In his session at 19th Cloud Expo, David Tishgart, Director of Product Marketing ...
Sep. 26, 2016 05:00 PM EDT Reads: 1,827
SYS-CON Events announced today that Bsquare has been named “Silver Sponsor” of SYS-CON's @ThingsExpo, which will take place on November 1–3, 2016, at the Santa Clara Convention Center in Santa Clara, CA. For more than two decades, Bsquare has helped its customers extract business value from a broad array of physical assets by making them intelligent, connecting them, and using the data they generate to optimize business processes.
Sep. 26, 2016 05:00 PM EDT Reads: 2,717
In his keynote at 18th Cloud Expo, Andrew Keys, Co-Founder of ConsenSys Enterprise, provided an overview of the evolution of the Internet and the Database and the future of their combination – the Blockchain. Andrew Keys is Co-Founder of ConsenSys Enterprise. He comes to ConsenSys Enterprise with capital markets, technology and entrepreneurial experience. Previously, he worked for UBS investment bank in equities analysis. Later, he was responsible for the creation and distribution of life sett...
Sep. 26, 2016 04:30 PM EDT Reads: 3,353
Digitization is driving a fundamental change in society that is transforming the way businesses work with their customers, their supply chains and their people. Digital transformation leverages DevOps best practices, such as Agile Parallel Development, Continuous Delivery and Agile Operations to capitalize on opportunities and create competitive differentiation in the application economy. However, information security has been notably absent from the DevOps movement. Speed doesn’t have to negat...
Sep. 26, 2016 04:30 PM EDT Reads: 2,100
If you’re responsible for an application that depends on the data or functionality of various IoT endpoints – either sensors or devices – your brand reputation depends on the security, reliability, and compliance of its many integrated parts. If your application fails to deliver the expected business results, your customers and partners won't care if that failure stems from the code you developed or from a component that you integrated. What can you do to ensure that the endpoints work as expect...
Sep. 26, 2016 04:30 PM EDT Reads: 1,616
The Transparent Cloud-computing Consortium (abbreviation: T-Cloud Consortium) will conduct research activities into changes in the computing model as a result of collaboration between "device" and "cloud" and the creation of new value and markets through organic data processing High speed and high quality networks, and dramatic improvements in computer processing capabilities, have greatly changed the nature of applications and made the storing and processing of data on the network commonplace.
Sep. 26, 2016 04:15 PM EDT Reads: 1,025
Your business relies on your applications and your employees to stay in business. Whether you develop apps or manage business critical apps that help fuel your business, what happens when users experience sluggish performance? You and all technical teams across the organization – application, network, operations, among others, as well as, those outside the organization, like ISPs and third-party providers – are called in to solve the problem.
Sep. 26, 2016 04:00 PM EDT Reads: 2,524
SYS-CON Events announced today that ReadyTalk, a leading provider of online conferencing and webinar services, has been named Vendor Presentation Sponsor at the 19th International Cloud Expo, which will take place on November 1–3, 2016, at the Santa Clara Convention Center in Santa Clara, CA. ReadyTalk delivers audio and web conferencing services that inspire collaboration and enable the Future of Work for today’s increasingly digital and mobile workforce. By combining intuitive, innovative tec...
Sep. 26, 2016 04:00 PM EDT Reads: 2,839
Cognitive Computing is becoming the foundation for a new generation of solutions that have the potential to transform business. Unlike traditional approaches to building solutions, a cognitive computing approach allows the data to help determine the way applications are designed. This contrasts with conventional software development that begins with defining logic based on the current way a business operates. In her session at 18th Cloud Expo, Judith S. Hurwitz, President and CEO of Hurwitz & ...
Sep. 26, 2016 03:45 PM EDT Reads: 2,912
The vision of a connected smart home is becoming reality with the application of integrated wireless technologies in devices and appliances. The use of standardized and TCP/IP networked wireless technologies in line-powered and battery operated sensors and controls has led to the adoption of radios in the 2.4GHz band, including Wi-Fi, BT/BLE and 802.15.4 applied ZigBee and Thread. This is driving the need for robust wireless coexistence for multiple radios to ensure throughput performance and th...
Sep. 26, 2016 03:30 PM EDT Reads: 1,557
Enterprise IT has been in the era of Hybrid Cloud for some time now. But it seems most conversations about Hybrid are focused on integrating AWS, Microsoft Azure, or Google ECM into existing on-premises systems. Where is all the Private Cloud? What do technology providers need to do to make their offerings more compelling? How should enterprise IT executives and buyers define their focus, needs, and roadmap, and communicate that clearly to the providers?
Sep. 26, 2016 03:00 PM EDT Reads: 1,556