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." (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - July 15, 2014 Category: Neurology Source Type: news
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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - July 15, 2014 Category: Neurology Source Type: news
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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - July 15, 2014 Category: Neurology Source Type: news
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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - July 2, 2014 Category: Neurology Source Type: news
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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - June 30, 2014 Category: Neurology Source Type: news
