BioMarin Discontinues Clinical Development of Drisapersen
BioMarin has announced that they were discontinuing the clinical development of drisapersen after interactions with both the FDA and EMA. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - June 1, 2016 Category: Neurology Source Type: news
PPMD ’s Every Single One Tour: Salt Lake City
With a successful Every Single One tour stop in Baltimore under our belts, PPMD visited Salt Lake City this past weekend. Thank you to all of the families from across Utah and Idaho who joined us! < div class="feedflare" > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=o2il7igkY4k:XDLY3ARnYBE:yIl2AUoC8zA" > < img src="http://feeds.feedburner.com/~ff/ppmd?d=yIl2AUoC8zA" border="0" > < /img > < /a > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=o2il7igkY4k:XDLY3ARnYBE:qj6IDK7rITs" > < img src="http://feeds.feedburner.com/~ff/ppmd?d=qj6IDK7rITs" border="0" > < /img > < /a > < a href="http://feeds.parentpr...
Source: Parent Project Muscular Dystrophy - May 26, 2016 Category: Neurology Source Type: news
PPMD’s Every Single One Tour: Salt Lake City
With a successful Every Single One tour stop in Baltimore under our belts, PPMD visited Salt Lake City this past weekend. Thank you to all of the families from across Utah and Idaho who joined us! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 26, 2016 Category: Neurology Source Type: news
Sarepta Announces FDA Will Not Complete the Review of the Eteplirsen NDA By The PDUFA Date
The FDA has notified Sarepta that they are continuing their review of eteplirsen and will not be able to complete their work by the PDUFA goal date of May 26. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 25, 2016 Category: Neurology Source Type: news
[Webinar Recording] MissionDMD: FibroGen ’s Anti-Fibrosis Program
PPMD and FibroGen recently hosted a webinar to discuss the launch of FibroGen's MissionDMD program that is being conducted to investigate FG-3019 in Duchenne. < div class="feedflare" > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=NhLV8ajewn4:ruQX-IyYYhU:yIl2AUoC8zA" > < img src="http://feeds.feedburner.com/~ff/ppmd?d=yIl2AUoC8zA" border="0" > < /img > < /a > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=NhLV8ajewn4:ruQX-IyYYhU:qj6IDK7rITs" > < img src="http://feeds.feedburner.com/~ff/ppmd?d=qj6IDK7rITs" border="0" > < /img > < /a > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=NhLV8ajewn4:ruQX-IyYY...
Source: Parent Project Muscular Dystrophy - May 20, 2016 Category: Neurology Source Type: news
[Webinar Recording] MissionDMD: FibroGen’s Anti-Fibrosis Program
PPMD and FibroGen recently hosted a webinar to discuss the launch of FibroGen's MissionDMD program that is being conducted to investigate FG-3019 in Duchenne. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 20, 2016 Category: Neurology Source Type: news
Next Steps with Newborn Screening
PPMD is leading a national effort including more than 50 of the world’s top newborn screening and Duchenne experts to build a newborn screening infrastructure for Duchenne in the United States. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 19, 2016 Category: Neurology Source Type: news
DRSC Definitions
PPMD is undertaking multiple efforts to help improve clinical trial design in order to accelerate clinical trials and find promising therapies for Duchenne. But how does data aggregation really work? Our friends at D-RSC have spelled out some concepts and terms for you to make understanding all that PPMD is doing easier. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 17, 2016 Category: Neurology Source Type: news
PPMD Names Children ’s Mercy Hospital in Kansas City, MO, Certified Duchenne Care Center
Children ’s Mercy Hospital in Kansas City, Missouri became the 11th clinic named a Certified Duchenne Care Center by PPMD. < div class="feedflare" > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=HDzdtYwPtl8:Rx0FHRzMASI:yIl2AUoC8zA" > < img src="http://feeds.feedburner.com/~ff/ppmd?d=yIl2AUoC8zA" border="0" > < /img > < /a > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=HDzdtYwPtl8:Rx0FHRzMASI:qj6IDK7rITs" > < img src="http://feeds.feedburner.com/~ff/ppmd?d=qj6IDK7rITs" border="0" > < /img > < /a > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=HDzdtYwPtl8:Rx0FHRzMASI:V_sGLiPBpWU" > < img src="http:...
Source: Parent Project Muscular Dystrophy - May 16, 2016 Category: Neurology Source Type: news
PPMD Names Children’s Mercy Hospital in Kansas City, MO, Certified Duchenne Care Center
Children’s Mercy Hospital in Kansas City, Missouri became the 11th clinic named a Certified Duchenne Care Center by PPMD. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 16, 2016 Category: Neurology Source Type: news
End Duchenne eNews: Every Single Carrier
Catch up on the latest research and community updates in this month's End Duchenne eNews. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 16, 2016 Category: Neurology Source Type: news
PPMD Brings Together Experts to Assess Steroid Effects on Bone Health in Duchenne
More than 50 international experts in both pediatric and Duchenne bone health have gathered to attend PPMD’s Bone Health and Osteoporosis Workshop today & tomorrow in order to dive deeply into these important topics. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 12, 2016 Category: Neurology Source Type: news
Every Single Carrier
PPMD is proud to formally announce our carrier initiative. Like everything we do, PPMD will look at carrier issues from a variety of angles, through a variety of lenses. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 9, 2016 Category: Neurology Source Type: news
Changing the Paradigm: The Eteplirsen Ad Comm
There is no way around it, today was hard. Like you, we are dumbfounded by the closing votes and discussion. But a decision has not been made – these were recommendations. And we believe that the number of questions with mixed votes represent opportunities for us to change minds and continue to influence the agency. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - April 26, 2016 Category: Neurology Source Type: news
April 25: FDA Advisory Committee Meeting for Eteplirsen - Live Webcast Information
The Center for Drug Evaluation and Research (CDER) plans to provide a free of charge, live webcast of the meeting. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - April 22, 2016 Category: Neurology Source Type: news
