Parent Project Muscular Dystrophy Endorses FAST Act Legislation to Expedite FDA Review... -- HACKENSACK, N.J., March 29, 2012 /PRNewswire-USNewswire/ --

Parent Project Muscular Dystrophy Endorses FAST Act Legislation to Expedite FDA Review... -- HACKENSACK, N.J., March 29, 2012 /PRNewswire-USNewswire/ --: HACKENSACK, N.J., March 29, 2012 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) – the leading advocacy organization fighting to end Duchenne muscular dystrophy – has endorsed legislation that would shorten the amount of time the Food and Drug Administration (FDA) takes to review candidate therapies for life-threatening conditions like Duchenne that lack other treatment options.

"The FAST Act is commonsense legislation that would leverage the Fast Track and Accelerated Review options at FDA's disposal. It would allow sponsors of candidate drugs to petition FDA to designate their product as being eligible for Fast Track review. It would also allow for approval of therapies on the condition of surrogate endpoints or intermediate markers used to gauge the trajectory of a disease," Furlong added.

Surrogate endpoints are a particularly important issue to the Duchenne community given the course of the disease and the need to more quickly determine whether or not a therapy is having its intended effect.

The legislation also seeks to more appropriately balance the benefits of a therapy with the potential risks. It would revoke accelerated approval if a sponsor fails to adequately study and monitor the therapy in patients or if further studies fail to verify clinical benefit.

"With nearly 20 potential therapies in clinical testing, it is a very exciting time for the Duchenne community, but a time in which we must make sure the FDA is equipped with the tools and laws it needs to evaluate these therapies as quickly as possible to save our sons," Furlong said.

"I commend Congressmen Stearns and Towns for introducing this legislation, and I urge other members to cosponsor it today so it can become law this year," she added.

Prosensa’s Clinical Development Plan Update – March 2012

In parallel to the on-going development of the lead compound PRO051/GSK2402968, which is being developed by GSK with an active involvement of Prosensa, there are five more compounds for Duchenne in Prosensa’s pipeline. Below you can find an update about the current state of development of these compounds.


http://community.parentprojectmd.org/profiles/blogs/prosensa-clinical-development-plan-update-march-2012?commentId=1187424%3AComment%3A150116&xg_source=msg_com_blogpost

Steroids: A Certain Can of Worms (A Complicated State of Affairs) - PPMD Community

Steroids: A Certain Can of Worms (A Complicated State of Affairs) - PPMD Community: Steroids are a complicated story and have clearly changed the natural history of Duchenne. We know they work for some, for most in fact, but not all. We know they increase strength and prolong ambulation, and statistics back this up. Predicting ambulation is prolonged on average for 2 years. But this is not the end of the story. Genetic modifiers have been identified that are thought to dramatically push out this timeline. Individuals with specific genetic modifiers continue walking into their mid- to late teens, often called outliers.

ImagingDMD MRI Study (DuchenneConnect Webinar Series) - PPMD Community

ImagingDMD MRI Study (DuchenneConnect Webinar Series) - PPMD Community: Dr. Krista Vandenborne and Claudia Senesac, PT, PhD, PCS, both from the University of Florida, discuss the ImagingDMD Study. This study focuses on developing Magnetic Resonance Imaging (MRI) as a tool to monitor disease progression in Duchenne and to serve as an outcome measure for clinical trials.

The aim of the study is to determine whether noninvasive MRI outcome measures can replace muscle biopsies in evaluating the effectiveness of new treatments in future clinical trials. A second goal of this study is to characterize the progressive involvement of the lower extremity muscles to help guide the design of future clinical trials.

Important Webinar: AVI Clinical Trial Update - PPMD Community

Important Webinar: AVI Clinical Trial Update - PPMD Community: Parent Project Muscular Dystrophy presents an AVI webinar on Wednesday, March 21 at 1pm eastern. The webinar will cover a pre-data update on AVI BioPharma's Phase IIb study evaluating eteplirsen for the treatment of Duchenne patients with mutations amenable to exon-51 skipping, and will be lead by Chris Garabedian, the CEO of AVI BioPharma.

New three-step process for DMD newborn screening

New three-step process for DMD newborn screening: Investigators at Nationwide Children's Hospital, working with the DNA Sequencing Core Facility at the University of Utah, have developed an approach to newborn screening (NBS) for the life-threatening genetic disorder, Duchenne muscular dystrophy (DMD) and potentially other muscular dystrophies.

Action Duchenne’s Seeds of Hope Appeal set to blossom for the third successive year - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Action Duchenne’s Seeds of Hope Appeal set to blossom for the third successive year - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "National sunflower campaign encourages children to learn about the environment and growing plants while raising awareness and funds for charity Action Duchenne

ARTICLE POSTED: 14 MARCH 2012 PERMANENT LINK
Action Duchenne is launching its national campaign, the Seeds of Hope Appeal for the third year running. Seeds of Hope is aimed at children between the ages of 3 and 11 years old, with the objective to raise awareness of the life limiting condition Duchenne Muscular Dystrophy. The appeal, which has been very successful in the last two years with over 3,000 participants last year, will raise funds for the charity, which campaigns to find a cure and viable treatments for Duchenne Muscular Dystrophy. "

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