PPMD's Pat Furlong on BioCentury This Week
Thank you "BioCentury This Week" for having PPMD Founding President & CEO Pat Furlong on its 200th episode this weekend! You can watch Pat's segment online, where she shares her personal story in founding PPMD and why we will not rest until every person has a treatment to end Duchenne.
No Exon Left Behind
PPMD has a decade-long history of supporting exon-skipping, but what exactly is it? How can we bring the technology to less common deletions and duplications as quickly as possible?
Action Alert! Key Senate Committee Schedules Markup for MD-CARE Act
In a significant step forward, a key Senate committee has announced that it will consider the MD-CARE Act Amendments on Wednesday, July 23rd. We need all members of the Senate to know how important advancing this bill is to our community. Please take a moment to email your Senators and ask your friends and family to do the same.
End Duchenne eNews: Awarding Grants, Recognizing Progress, Welcoming Leadership
Catch up on the latest research and community updates in this month's End Duchenne eNews.
One Step Closer
Progress! Today the House Energy & Commerce committee voted unanimously to advance H.R. 594, the MD-CARE Act Amendments of 2014. This action moves the bill to consideration and hopefully passage by the full House of Representatives.
PPMD President Testifies Before Key Congressional Committee
Pat Furlong, Founding President and CEO of Parent Project Muscular Dystrophy will testify before the House Energy and Commerce Committee’s Subcommittee on Health as part of the committee’s ongoing 21st Century Cures Initiative on July 11 at 9am eastern. She is one of four patient advocacy representatives selected to testify in a hearing entitled “21st Century Cures: Incorporating the Patient Perspective."
PPMD and NHLBI Co-Sponsor Duchenne Cardiac Working Group
PPMD is thrilled to partner with the National Heart, Lung, and Blood Institute (NHLBI) to convene a working group titled “Contemporary Cardiac Issues in Duchenne Muscular Dystrophy.” The group, made up of top pediatric and adult cardiologists as well as clinical researchers, will meet this week to address critical evidence gaps in the understanding of cardiac issues in Duchenne.
Outcomes Meeting: Laying a Foundation for Outcome Measures
At PPMD's pre-conference meeting, a group of therapists and industry representatives met to discuss the importance of standardizing outcome measures.
Conference 20 Recap
Conference 20 included young men, parents, medical experts, academic researchers and companies, more than 16 companies talking about current trials or trials in development. We discussed compounds that target dystrophin (exon skipping and nonsense suppression) as well as compounds that ‘chip’ away at the downstream pathology- steroid replacement, anti-myostatin, anti-inflammatories, anti-fibrotics as well as new targets.
PPMD Welcomes John Porter!
John Porter, PhD is a Program Director at the NIH/NINDS, responsible for managing a portfolio of research grants across neuromuscular disorders. John has agreed to join PPMD officially as of January 2015, to help us build upon current programs, expand the pipeline, and accelerate progress and approvals.
PPMD Submits FDA Draft Guidance on Duchenne
Today is a landmark day for PPMD and the entire Duchenne community. We are so pleased that at our organization’s 20 year anniversary after 2 decades of hard work and resolute persistence within the policy, legislative and regulatory arena we are today able to provide to the FDA the first-ever patient-initiated guidance to help accelerate development and review of potential therapies for Duchenne.
The best part of the Connect Conference is being there, being together, connecting with the Duchenne community, the annual reunion. HOPE and CHANGE filled the air.
July 2, 2014 | Read more