Welcome!

News Feed Item

Top Scientists and Mothers Appeal to Congress for Breakthrough Child-Saving Therapy

Call on FDA for Accelerated Approval of Promising Duchenne Muscular Dystrophy Treatment

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.

(Photo: http://photos.prnewswire.com/prnh/20140210/MN62028)

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

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
WebRTC is great technology to build your own communication tools. It will be even more exciting experience it with advanced devices, such as a 360 Camera, 360 microphone, and a depth sensor camera. In his session at @ThingsExpo, Masashi Ganeko, a manager at INFOCOM Corporation, will introduce two experimental projects from his team and what they learned from them. "Shotoku Tamago" uses the robot audition software HARK to track speakers in 360 video of a remote party. "Virtual Teleport" uses a...
SYS-CON Events announced today that Datera will exhibit at 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. Datera offers a radically new approach to data management, where innovative software makes data infrastructure invisible, elastic and able to perform at the highest level. It eliminates hardware lock-in and gives IT organizations the choice to source x86 server nodes, with business model option...
When shopping for a new data processing platform for IoT solutions, many development teams want to be able to test-drive options before making a choice. Yet when evaluating an IoT solution, it’s simply not feasible to do so at scale with physical devices. Building a sensor simulator is the next best choice; however, generating a realistic simulation at very high TPS with ease of configurability is a formidable challenge. When dealing with multiple application or transport protocols, you would be...
With 10 simultaneous tracks, keynotes, general sessions and targeted breakout classes, Cloud Expo and @ThingsExpo are two of the most important technology events of the year. Since its launch over eight years ago, Cloud Expo and @ThingsExpo have presented a rock star faculty as well as showcased hundreds of sponsors and exhibitors! In this blog post, I provide 7 tips on how, as part of our world-class faculty, you can deliver one of the most popular sessions at our events. But before reading the...
In his session at 21st Cloud Expo, Carl J. Levine, Senior Technical Evangelist for NS1, will objectively discuss how DNS is used to solve Digital Transformation challenges in large SaaS applications, CDNs, AdTech platforms, and other demanding use cases. Carl J. Levine is the Senior Technical Evangelist for NS1. A veteran of the Internet Infrastructure space, he has over a decade of experience with startups, networking protocols and Internet infrastructure, combined with the unique ability to it...
Internet of @ThingsExpo, taking place October 31 - November 2, 2017, at the Santa Clara Convention Center in Santa Clara, CA, is co-located with 21st Cloud Expo and will feature technical sessions from a rock star conference faculty and the leading industry players in the world. The Internet of Things (IoT) is the most profound change in personal and enterprise IT since the creation of the Worldwide Web more than 20 years ago. All major researchers estimate there will be tens of billions devic...
Historically, some banking activities such as trading have been relying heavily on analytics and cutting edge algorithmic tools. The coming of age of powerful data analytics solutions combined with the development of intelligent algorithms have created new opportunities for financial institutions. In his session at 20th Cloud Expo, Sebastien Meunier, Head of Digital for North America at Chappuis Halder & Co., discussed how these tools can be leveraged to develop a lasting competitive advantage ...
SYS-CON Events announced today that CA Technologies has been named "Platinum Sponsor" of SYS-CON's 21st International Cloud Expo®, which will take place October 31-November 2, 2017, at the Santa Clara Convention Center in Santa Clara, CA. CA Technologies helps customers succeed in a future where every business - from apparel to energy - is being rewritten by software. From planning to development to management to security, CA creates software that fuels transformation for companies in the applic...
As DevOps methodologies expand their reach across the enterprise, organizations face the daunting challenge of adapting related cloud strategies to ensure optimal alignment, from managing complexity to ensuring proper governance. How can culture, automation, legacy apps and even budget be reexamined to enable this ongoing shift within the modern software factory?
SYS-CON Events announced today that Elastifile will exhibit at 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. Elastifile Cloud File System (ECFS) is software-defined data infrastructure designed for seamless and efficient management of dynamic workloads across heterogeneous environments. Elastifile provides the architecture needed to optimize your hybrid cloud environment, by facilitating efficient...
“Why didn’t testing catch this” must become “How did this make it to testing?” Traditional quality teams are the crutch and excuse keeping organizations from making the necessary investment in people, process, and technology to accelerate test automation. Just like societies that did not build waterways because the labor to keep carrying the water was so cheap, we have created disincentives to automate. In her session at @DevOpsSummit at 20th Cloud Expo, Anne Hungate, President of Daring System...
SYS-CON Events announced today that GrapeUp, the leading provider of rapid product development at the speed of business, will exhibit at SYS-CON's 21st International Cloud Expo®, which will take place October 31-November 2, 2017, at the Santa Clara Convention Center in Santa Clara, CA. Grape Up is a software company, specialized in cloud native application development and professional services related to Cloud Foundry PaaS. With five expert teams that operate in various sectors of the market acr...
Most companies are adopting or evaluating container technology - Docker in particular - to speed up application deployment, drive down cost, ease management and make application delivery more flexible overall. As with most new architectures, this dream takes a lot of work to become a reality. Even when you do get your application componentized enough and packaged properly, there are still challenges for DevOps teams to making the shift to continuous delivery and achieving that reduction in cost ...
"Cloud computing is certainly changing how people consume storage, how they use it, and what they use it for. It's also making people rethink how they architect their environment," stated Brad Winett, Senior Technologist for DDN Storage, in this SYS-CON.tv interview at 20th Cloud Expo, held June 6-8, 2017, at the Javits Center in New York City, NY.
"We provide IoT solutions. We provide the most compatible solutions for many applications. Our solutions are industry agnostic and also protocol agnostic," explained Richard Han, Head of Sales and Marketing and Engineering at Systena America, in this SYS-CON.tv interview at @ThingsExpo, held June 6-8, 2017, at the Javits Center in New York City, NY.