[Webinar Recording] NS Pharma's Exon 53 Skipping Program
PPMD recently hosted webinar with NS Pharma (US subsidiary of Nippon Shinyaku, Co.,Ltd) to discuss NS Pharma's Exon 53 Skipping Program. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - November 22, 2016 Category: Neurology Source Type: news
Action Alert: Tell Congress to Pass 21st Century Cures!
On November 15th, rare disease advocates from across the country will be flooding Congressional inboxes and phone lines with one message: Pass 21st Century Cures in this Congress. Please consider taking action Tuesday and all week! Otherwise the work we ’ve done over the past two years will be lost and the process will begin again in a new Congress. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - November 18, 2016 Category: Neurology Source Type: news
PPMD Awards $239,000 Research Grant to Dr. Terence Partridge of Children ’s National Medical Center
(Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - November 18, 2016 Category: Neurology Source Type: news
Calling All Adults with Duchenne/Becker – Join the 2017 PPMD Adult Advisory Committee (PAAC)!
(Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - November 18, 2016 Category: Neurology Source Type: news
[Webinar Takeaways] Preparing for Access to Approved Therapies
(Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - November 18, 2016 Category: Neurology Source Type: news
Anthem and Exondys 51 (Eteplirsen)
(Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - November 18, 2016 Category: Neurology Source Type: news
[Webinar Recording] Deflazacort Access: Navigating Potential Changes to Your Prescription
PPMD recently hosted a webinar with Masters Pharmaceuticals to discuss the transition from Masters ’ deflazacort to Marathon Pharmaceuticals deflazacort, should Marathon receive FDA approval in the U.S. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - November 18, 2016 Category: Neurology Source Type: news
Duchenne Carrier Study at Nationwide Children ’s Hospital
PPMD is excited to support this important study that will evaluate the effects of a lower level of dystrophin in female carriers. We hope the results of this study will help us better understand the implications of being a Duchenne carrier, and possibly apply some of the answers to our understanding of Duchenne in males. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - November 14, 2016 Category: Neurology Source Type: news
Registration for the 2017 Bank of America Chicago Marathon is now open!
Join PPMD's Run For Our Sons team for the 40th anniversary of this incredible race, on Sunday, November 4, 2017. Our entries are only available until January 18th, so sign up soon! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - November 2, 2016 Category: Neurology Source Type: news
Understanding Insurance Determinations
PPMD ’s Annie Kennedy provides an update on our Duchenne community’s current access environment, and actions that those who are amenable to exon 51 skipping can take in order to encourage their providers to cover EXONDYS 51. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - November 2, 2016 Category: Neurology Source Type: news
Deflazacort Access: Navigating Potential Changes to Your Prescription
PPMD has put together a resource we hope will answer some of the questions that have arisen regarding the potential FDA approval of deflazacort in the U.S. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - October 31, 2016 Category: Neurology Source Type: news
The Assistance Fund Launches Duchenne Program to Support Access to Exondys 51
The Assistance Fund has launched a new program in Duchenne to support access to Exondys 51 after efforts through SareptAssist have been other-wise exhausted. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - October 28, 2016 Category: Neurology Source Type: news
PPMD Designates Center for Duchenne Muscular Dystrophy at UCLA, a Certified Duchenne Care Center
Our Duchenne families in Los Angeles are extremely fortunate to have this team of dedicated clinicians and researchers at UCLA. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - October 26, 2016 Category: Neurology Source Type: news
The Latest Update on Translarna
PPMD continues to believe that our Duchenne community deserves a full and fair review of the Translarna data by scientific experts at an Advisory Committee Meeting. If you have any experience on Translarna, or are waiting for access to Translarna, please share your experience with us so that we can translate your story into rigorous data that can be shared with FDA. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - October 26, 2016 Category: Neurology Source Type: news
Register for PPMD's 2017 Advocacy Conference
Your presence in Washington, DC this February is a critical part of the Duchenne community's strength and impact going forward. Please consider joining us February 12-14 as we urge Congress to continue to make Duchenne a priority! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - October 20, 2016 Category: Neurology Source Type: news
