Update on the 2015 Flu
Even after receiving vaccination, many people are still contracting the flu this season. Read an update on the latest flu strains as well as important information on how to treat the flu if you or your child has Duchenne. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - January 20, 2015 Category: Neurology Source Type: news
PPMD Welcomes New CEO, John Porter, PhD
PPMD is thrilled to welcome our new CEO, Dr. John Porter! As CEO, John will help PPMD continue to integrate programs to maximize impact, building upon current programs and exploring opportunities to expand the research pipeline, with the goal of accelerating progress and approvals. John joins PPMD Founding President Pat Furlong and Chief Operating Officer, Kimberly Galberaith, in leading the organization. As founding president, Pat will continue to provide strategic direction of PPMD in support of the mission she helped set forth 20 years ago in honor of her sons and all families affected by Duchenne. (Source: Parent Proje...
Source: Parent Project Muscular Dystrophy - January 20, 2015 Category: Neurology Source Type: news
Meet Our 2015 PPMD Adult Advisory Council
The PPMD Adult Advisory Council (PAAC) serves to ensure that the voices of adults living with Duchenne and Becker muscular dystrophy are reflected in PPMD’s legislative, policy, and program activities. PAAC members serve in a formal advisory capacity for their respective term and maintain leadership over a PAAC initiative throughout that term; PAAC initiatives are determined by the PAAC membership and PPMD Executive Team. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - January 20, 2015 Category: Neurology Source Type: news
PPMD Awards Akashi Therapeutics, Inc. $500,000 Grant
Parent Project Muscular Dystrophy (PPMD) has awarded Akashi Therapeutics, Inc. (Akashi) a $500,000 grant to fund clinical trials to test the safety and efficacy of an investigational new drug known as HT-100 (delayed-release halofuginone).
HT-100 is an orally available small molecule drug candidate being developed to reduce fibrosis and inflammation and to promote healthy muscle fiber regeneration in Duchenne patients. The application of HT-100 to Duchenne and other fibrotic diseases is based on pioneering work by Dr. Mark Pines at the Volcani Institute in Israel. Akashi, led by CEO Marc Blaustein, has been granted orphan ...
Source: Parent Project Muscular Dystrophy - December 8, 2014 Category: Neurology Source Type: news
PPMD Calls for Greater Transparency and Accountability of FDA Patient Engagement Efforts
Parent Project Muscular Dystrophy (PPMD) is urging Congress to build upon gains in recent years to further strengthen and enhance programs to ensure the patient voice is heeded during the drug development process during a Congressional briefing tomorrow, Tuesday, December 9th at noon.
A champion of policies to enhance the voice of the patient within vital Food and Drug Administration (FDA) deliberations, PPMD is advocating for provisions within the 21st Century Cures Initiatives focused on measuring how the agency is or is not using new patient engagement tools. PPMD and other stakeholders will be presenting on the refor...
Source: Parent Project Muscular Dystrophy - December 8, 2014 Category: Neurology Source Type: news
ABLE Act Changes Disability Landscape –
This just got real!
Thank you to the House of Representatives for voting in favor of civil rights for Americans with disabilities today by passing the ABLE Act (S. 313/ HR 647)! With legendary bipartisan support in both the House and Senate, the ABLE Act amends Section 529 of the Internal Revenue Service Code of 1986 to create tax-free savings accounts for individuals with disabilities. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - December 8, 2014 Category: Neurology Source Type: news
Webinar Recording & Summary: Duchenne Pulmonary Care for Adults
A summary and recording of last week's pulmonary care webinar is now available. Thank you to Dr. Daniel Sheehan and Dr. Lisa Wolfe for such an informative discussion. < div class="feedflare" > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=wxXKXf9v9k4:TcP0GqwKbvg:yIl2AUoC8zA" > < img src="http://feeds.feedburner.com/~ff/ppmd?d=yIl2AUoC8zA" border="0" > < /img > < /a > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=wxXKXf9v9k4:TcP0GqwKbvg:qj6IDK7rITs" > < img src="http://feeds.feedburner.com/~ff/ppmd?d=qj6IDK7rITs" border="0" > < /img > < /a > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=wxXKXf9v9k4:T...
Source: Parent Project Muscular Dystrophy - December 8, 2014 Category: Neurology Source Type: news
Webinar Recording & Summary: Duchenne Pulmonary Care for Adults
A summary and recording of last week's pulmonary care webinar is now available. Thank you to Dr. Daniel Sheehan and Dr. Lisa Wolfe for such an informative discussion. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - December 8, 2014 Category: Neurology Source Type: news
Combine therapies. Combine forces.
In 2015, PPMD will invest $600,000 to further the advancement of combination therapies in Duchenne. To start, we need to raise $400,000 this holiday season. We know that sounds big, but thanks to our partners, it’s within reach—every gift today goes twice as far thanks to two Duchenne foundations that, like us, believe in the importance of combined therapies to treat this horrible disease. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - December 1, 2014 Category: Neurology Source Type: news
PPMD Names Baltimore ’s Kennedy Krieger Institute a Certified Duchenne Care Center
Kennedy Krieger Institute in Baltimore, Maryland was named a Certified Duchenne Care Center by Parent Project Muscular Dystrophy (PPMD), the leading advocacy organization working to end Duchenne muscular dystrophy (Duchenne). Kennedy Krieger Institute is the fifth center to be certified by PPMD, recognizing the Institute ’s dedication to improving care for people living with Duchenne. < div class="feedflare" > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=SSxSBN6ipWo:lCqOGd6-Fuw:yIl2AUoC8zA" > < img src="http://feeds.feedburner.com/~ff/ppmd?d=yIl2AUoC8zA" border="0" > < /img > < /a > < a href="http://feeds.parent...
Source: Parent Project Muscular Dystrophy - November 20, 2014 Category: Neurology Source Type: news
PPMD Names Baltimore’s Kennedy Krieger Institute a Certified Duchenne Care Center
Kennedy Krieger Institute in Baltimore, Maryland was named a Certified Duchenne Care Center by Parent Project Muscular Dystrophy (PPMD), the leading advocacy organization working to end Duchenne muscular dystrophy (Duchenne). Kennedy Krieger Institute is the fifth center to be certified by PPMD, recognizing the Institute’s dedication to improving care for people living with Duchenne. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - November 20, 2014 Category: Neurology Source Type: news
Patients Are Waiting...
The FDA is listening and has told us as much. But that doesn’t mean we should stop doing everything we can to educate the Agency.
Thanks to the dozens of you who participated in PPMD’s benefit/risk project and took the time to share your story, we have been able to compile your responses and will be providing the FDA with “Patients Are Waiting…” (download PDF). Collected on our community site, we have referenced these stories in our interactions with the FDA over the last several months but we wanted them to really understand the urgency of our collective voice, the volume of our collective cries.
These are you...
Source: Parent Project Muscular Dystrophy - November 4, 2014 Category: Neurology Source Type: news
PPMD Submits Letter to FDA Regarding Latest Sarepta Update
Today’s press release and investor call from Sarepta have sent a ripple of angst and sadness through our community. Moments ago, PPMD sent a letter to the FDA that reflects our organization’s belief that safety and rigor have been demonstrated throughout this process. Further, we believe that any strengthening of data packages that causes delays (as is now being requested by the FDA, as articulated in today’s press release and call) should ensure a streamlined, more expeditious approval process for follow-on exons.
As a community we believe exon-skipping drugs like eteplirsen hold potentially life-altering benefits...
Source: Parent Project Muscular Dystrophy - October 27, 2014 Category: Neurology Source Type: news
PPMD-Funded Gene Therapy Study Improves Walking Ability in Muscular Dystrophy
Dr. Jerry Mendell of Nationwide Children’s Research Institute recently presented data from a ground-breaking PPMD-funded study demonstrating that a modified virus carrying the gene for follistatin can improve performance on the 6 minute walk test in study participants with Becker muscular dystrophy. This is the first time a gene therapy has demonstrated an improvement in function in a muscular dystrophy. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - October 23, 2014 Category: Neurology Source Type: news
News from World Muscle Society Meeting (10/8/14)
Check out news from the 19th International World Muscle Society Congress in our latest blog: http://j.mp/1yPZtVp.
Topics covered:
► Highlights from Sarepta's symposium on the role of dystrophin production in disease-modifying treatments for Duchenne
► Becker muscular dystrophy updates
► Protecting bone health
► A “one stop” strategy to address mutations that occur before Exon 5
► Progress in skipping multiple exons
► A peak at new compounds
► Next generation utrophin upregulators
► Drisapersen long-term data (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - October 9, 2014 Category: Neurology Source Type: news
