|By PR Newswire||
|March 19, 2014 10:09 AM EDT||
WASHINGTON, March 19, 2014 /PRNewswire/ -- Parents of children facing devastating muscle wasting and premature death from Duchenne Muscular Dystrophy are calling on the U.S. Food and Drug Administration to accelerate approval of a new drug that leading scientists say is safe and effective.
Through their campaign "The Race to Yes," the families need 100,000 signatures by March 29 on a petition to guarantee a White House response. As of March 18, they have 43,750 signatures.
The parents believe the FDA is discounting clear evidence that a drug – eteplirsen – can safely and effectively treat the world's leading genetic killer of children. Long-term testing in boys with Duchenne amenable to this drug shows it halts progression of the disease and has no side effects.
"Every day that the FDA stalls, Duchenne takes children's lives," said Tracy Seckler whose son, Charley, has the disease. "The FDA claims this is a priority, but they've been sending us the same bureaucratic form letter for several months. If they grant accelerated approval, our children could be the first generation of Duchenne patients to survive."
Marissa Penrod, whose son Joseph also has Duchenne, said, "I've been praying for a miracle, and now that miracle is actually here in the form of a treatment that could pave the way to help all children with Duchenne. I'm begging President Obama to step in and hold the FDA accountable to follow the law and approve this drug."
More than 20 Duchenne-related organizations have joined the national grassroots campaign to get 100,000 signatures by the deadline.
Last month, seven of the world's leading Duchenne scientists and researchers met with FDA officials in Washington, D.C. to urge approval of the drug. Thirty-eight members of the U.S. House and Senate have written the FDA urging it to make greater use of a 2012 law that allows for accelerated approval for drugs to treat rare and fatal diseases that have no other treatments.
Australian professor Steve Wilton, a neuromuscular researcher and a pioneer in this science, told the FDA "in Australia we'd say it's bleeding obvious" that the drug works.
Celebrities such as Olympic gold medalist Aly Raisman, Dallas Mavericks owner Mark Cuban and actor/rapper LL Cool J have tweeted asking their followers to sign the petition.
Media Contact: Norris Clark, 609-425-1325
SOURCE The Race to Yes
There are so many tools and techniques for data analytics that even for a data scientist the choices, possible systems, and even the types of data can be daunting. In his session at @ThingsExpo, Chris Harrold, Global CTO for Big Data Solutions for EMC Corporation, will show how to perform a simple, but meaningful analysis of social sentiment data using freely available tools that take only minutes to download and install. Participants will get the download information, scripts, and complete en...
Oct. 13, 2015 03:00 AM EDT Reads: 399
Too often with compelling new technologies market participants become overly enamored with that attractiveness of the technology and neglect underlying business drivers. This tendency, what some call the “newest shiny object syndrome,” is understandable given that virtually all of us are heavily engaged in technology. But it is also mistaken. Without concrete business cases driving its deployment, IoT, like many other technologies before it, will fade into obscurity.
Oct. 13, 2015 03:00 AM EDT Reads: 255
Containers are changing the security landscape for software development and deployment. As with any security solutions, security approaches that work for developers, operations personnel and security professionals is a requirement. In his session at @DevOpsSummit, Kevin Gilpin, CTO and Co-Founder of Conjur, will discuss various security considerations for container-based infrastructure and related DevOps workflows.
Oct. 13, 2015 03:00 AM EDT Reads: 321
Containers are all the rage among developers and web companies, but they also represent two very substantial benefits to larger organizations. First, they have the potential to dramatically accelerate the application lifecycle from software builds and testing to deployment and upgrades. Second they represent the first truly hybrid-approach to consuming infrastructure, allowing organizations to run the same workloads on any cloud, virtual machine or physical server. Together, they represent a ver...
Oct. 13, 2015 02:45 AM EDT Reads: 200
SYS-CON Events announced today that Super Micro Computer, Inc., a global leader in high-performance, high-efficiency server, storage technology and green computing, will exhibit at the 17th International Cloud Expo®, which will take place on November 3–5, 2015, at the Santa Clara Convention Center in Santa Clara, CA. Supermicro (NASDAQ: SMCI), the leading innovator in high-performance, high-efficiency server technology is a premier provider of advanced server Building Block Solutions® for Data ...
Oct. 13, 2015 02:30 AM EDT Reads: 182
SYS-CON Events announced today the Containers & Microservices Bootcamp, being held November 3-4, 2015, in conjunction with 17th Cloud Expo, @ThingsExpo, and @DevOpsSummit at the Santa Clara Convention Center in Santa Clara, CA. This is your chance to get started with the latest technology in the industry. Combined with real-world scenarios and use cases, the Containers and Microservices Bootcamp, led by Janakiram MSV, a Microsoft Regional Director, will include presentations as well as hands-on...
Oct. 13, 2015 02:30 AM EDT Reads: 147
Between the compelling mockups and specs produced by analysts, and resulting applications built by developers, there exists a gulf where projects fail, costs spiral, and applications disappoint. Methodologies like Agile attempt to address this with intensified communication, with partial success but many limitations. In his session at DevOps Summit, Charles Kendrick, CTO and Chief Architect at Isomorphic Software, will present a revolutionary model enabled by new technologies. Learn how busine...
Oct. 13, 2015 02:00 AM EDT Reads: 388
Interested in leveraging automation technologies and a cloud architecture to make developers more productive? Learn how PaaS can benefit your organization to help you streamline your application development, allow you to use existing infrastructure and improve operational efficiencies. Begin charting your path to PaaS with OpenShift Enterprise.
Oct. 13, 2015 02:00 AM EDT Reads: 657
IT data is typically silo'd by the various tools in place. Unifying all the log, metric and event data in one analytics platform stops finger pointing and provides the end-to-end correlation. Logs, metrics and custom event data can be joined to tell the holistic story of your software and operations. For example, users can correlate code deploys to system performance to application error codes.
Oct. 13, 2015 02:00 AM EDT Reads: 313
Achim Weiss is Chief Executive Officer and co-founder of ProfitBricks. In 1995, he broke off his studies to co-found the web hosting company "Schlund+Partner." The company "Schlund+Partner" later became the 1&1 web hosting product line. From 1995 to 2008, he was the technical director for several important projects: the largest web hosting platform in the world, the second largest DSL platform, a video on-demand delivery network, the largest eMail backend in Europe, and a universal billing syste...
Oct. 13, 2015 02:00 AM EDT Reads: 348
Cloud computing delivers on-demand resources that provide businesses with flexibility and cost-savings. The challenge in moving workloads to the cloud has been the cost and complexity of ensuring the initial and ongoing security and regulatory (PCI, HIPAA, FFIEC) compliance across private and public clouds. Manual security compliance is slow, prone to human error, and represents over 50% of the cost of managing cloud applications. Determining how to automate cloud security compliance is critical...
Oct. 13, 2015 01:00 AM EDT Reads: 375
WebRTC services have already permeated corporate communications in the form of videoconferencing solutions. However, WebRTC has the potential of going beyond and catalyzing a new class of services providing more than calls with capabilities such as mass-scale real-time media broadcasting, enriched and augmented video, person-to-machine and machine-to-machine communications. In his session at @ThingsExpo, Luis Lopez, CEO of Kurento, will introduce the technologies required for implementing thes...
Oct. 13, 2015 12:45 AM EDT Reads: 849
Containers have changed the mind of IT in DevOps. They enable developers to work with dev, test, stage and production environments identically. Containers provide the right abstraction for microservices and many cloud platforms have integrated them into deployment pipelines. DevOps and containers together help companies achieve their business goals faster and more effectively. In his session at DevOps Summit, Ruslan Synytsky, CEO and Co-founder of Jelastic, will review the current landscape of...
Oct. 13, 2015 12:15 AM EDT Reads: 242
Electric power utilities face relentless pressure on their financial performance, and reducing distribution grid losses is one of the last untapped opportunities to meet their business goals. Combining IoT-enabled sensors and cloud-based data analytics, utilities now are able to find, quantify and reduce losses faster – and with a smaller IT footprint. Solutions exist using Internet-enabled sensors deployed temporarily at strategic locations within the distribution grid to measure actual line lo...
Oct. 13, 2015 12:00 AM EDT Reads: 273
“In the past year we've seen a lot of stabilization of WebRTC. You can now use it in production with a far greater degree of certainty. A lot of the real developments in the past year have been in things like the data channel, which will enable a whole new type of application," explained Peter Dunkley, Technical Director at Acision, in this SYS-CON.tv interview at @ThingsExpo, held Nov 4–6, 2014, at the Santa Clara Convention Center in Santa Clara, CA.
Oct. 12, 2015 11:15 PM EDT Reads: 7,123