Welcome!

News Feed Item

Children Dying While FDA Stalls on New Muscular Dystrophy Drug; Parents Seek 100,000 Signatures to Get White House's Attention

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.

www.TheRacetoYes.Org.

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

Logo - http://photos.prnewswire.com/prnh/20140311/MM81272LOGO

SOURCE The Race to Yes

More Stories By PR Newswire

Copyright © 2007 PR Newswire. All rights reserved. Republication or redistribution of PRNewswire content is expressly prohibited without the prior written consent of PRNewswire. PRNewswire shall not be liable for any errors or delays in the content, or for any actions taken in reliance thereon.

Latest Stories
While DevOps most critically and famously fosters collaboration, communication, and integration through cultural change, culture is more of an output than an input. In order to actively drive cultural evolution, organizations must make substantial organizational and process changes, and adopt new technologies, to encourage a DevOps culture. Moderated by Andi Mann, panelists discussed how to balance these three pillars of DevOps, where to focus attention (and resources), where organizations might...
No hype cycles or predictions of zillions of things here. IoT is big. You get it. You know your business and have great ideas for a business transformation strategy. What comes next? Time to make it happen. In his session at @ThingsExpo, Jay Mason, Associate Partner at M&S Consulting, presented a step-by-step plan to develop your technology implementation strategy. He discussed the evaluation of communication standards and IoT messaging protocols, data analytics considerations, edge-to-cloud tec...
When growing capacity and power in the data center, the architectural trade-offs between server scale-up vs. scale-out continue to be debated. Both approaches are valid: scale-out adds multiple, smaller servers running in a distributed computing model, while scale-up adds fewer, more powerful servers that are capable of running larger workloads. It’s worth noting that there are additional, unique advantages that scale-up architectures offer. One big advantage is large memory and compute capacity...
New competitors, disruptive technologies, and growing expectations are pushing every business to both adopt and deliver new digital services. This ‘Digital Transformation’ demands rapid delivery and continuous iteration of new competitive services via multiple channels, which in turn demands new service delivery techniques – including DevOps. In this power panel at @DevOpsSummit 20th Cloud Expo, moderated by DevOps Conference Co-Chair Andi Mann, panelists examined how DevOps helps to meet the de...
"When we talk about cloud without compromise what we're talking about is that when people think about 'I need the flexibility of the cloud' - it's the ability to create applications and run them in a cloud environment that's far more flexible,” explained Matthew Finnie, CTO of Interoute, in this SYS-CON.tv interview at 20th Cloud Expo, held June 6-8, 2017, at the Javits Center in New York City, NY.
The taxi industry never saw Uber coming. Startups are a threat to incumbents like never before, and a major enabler for startups is that they are instantly “cloud ready.” If innovation moves at the pace of IT, then your company is in trouble. Why? Because your data center will not keep up with frenetic pace AWS, Microsoft and Google are rolling out new capabilities. In his session at 20th Cloud Expo, Don Browning, VP of Cloud Architecture at Turner, posited that disruption is inevitable for comp...
SYS-CON Events announced today that Datanami has been named “Media Sponsor” of SYS-CON's 21st International Cloud Expo, which will take place on Oct 31 – Nov 2, 2017, at the Santa Clara Convention Center in Santa Clara, CA. Datanami is a communication channel dedicated to providing insight, analysis and up-to-the-minute information about emerging trends and solutions in Big Data. The publication sheds light on all cutting-edge technologies including networking, storage and applications, and thei...
"We are a monitoring company. We work with Salesforce, BBC, and quite a few other big logos. We basically provide monitoring for them, structure for their cloud services and we fit into the DevOps world" explained David Gildeh, Co-founder and CEO of Outlyer, in this SYS-CON.tv interview at DevOps Summit at 20th Cloud Expo, held June 6-8, 2017, at the Javits Center in New York City, NY.
SYS-CON Events announced today that Silicon India has been named “Media Sponsor” of SYS-CON's 21st International Cloud Expo, which will take place on Oct 31 – Nov 2, 2017, at the Santa Clara Convention Center in Santa Clara, CA. Published in Silicon Valley, Silicon India magazine is the premiere platform for CIOs to discuss their innovative enterprise solutions and allows IT vendors to learn about new solutions that can help grow their business.
Join us at Cloud Expo June 6-8 to find out how to securely connect your cloud app to any cloud or on-premises data source – without complex firewall changes. More users are demanding access to on-premises data from their cloud applications. It’s no longer a “nice-to-have” but an important differentiator that drives competitive advantages. It’s the new “must have” in the hybrid era. Users want capabilities that give them a unified view of the data to get closer to customers and grow business. The...
The Internet giants are fully embracing AI. All the services they offer to their customers are aimed at drawing a map of the world with the data they get. The AIs from these companies are used to build disruptive approaches that cannot be used by established enterprises, which are threatened by these disruptions. However, most leaders underestimate the effect this will have on their businesses. In his session at 21st Cloud Expo, Rene Buest, Director Market Research & Technology Evangelism at Ara...
"We do one of the best file systems in the world. We learned how to deal with Big Data many years ago and we implemented this knowledge into our software," explained Jakub Ratajczak, Business Development Manager at MooseFS, in this SYS-CON.tv interview at 20th Cloud Expo, held June 6-8, 2017, at the Javits Center in New York City, NY.
Five years ago development was seen as a dead-end career, now it’s anything but – with an explosion in mobile and IoT initiatives increasing the demand for skilled engineers. But apart from having a ready supply of great coders, what constitutes true ‘DevOps Royalty’? It’ll be the ability to craft resilient architectures, supportability, security everywhere across the software lifecycle. In his keynote at @DevOpsSummit at 20th Cloud Expo, Jeffrey Scheaffer, GM and SVP, Continuous Delivery Busine...
In his session at @ThingsExpo, Eric Lachapelle, CEO of the Professional Evaluation and Certification Board (PECB), provided an overview of various initiatives to certify the security of connected devices and future trends in ensuring public trust of IoT. Eric Lachapelle is the Chief Executive Officer of the Professional Evaluation and Certification Board (PECB), an international certification body. His role is to help companies and individuals to achieve professional, accredited and worldwide re...
SYS-CON Events announced today that TechTarget has been named “Media Sponsor” of SYS-CON's 21st International Cloud Expo, which will take place on Oct 31 – Nov 2, 2017, at the Santa Clara Convention Center in Santa Clara, CA. TechTarget storage websites are the best online information resource for news, tips and expert advice for the storage, backup and disaster recovery markets.