February 2017 Blog Posts (10)
Staff PPMD Joins Rare Disease Advocates in Letter to President
Added by Annie Kennedy on February 27, 2017 at 7:30pm — No Comments
Recap: PPMD's Adult Advisory Committee (PAAC) Leadership Meeting and 2017 Advocacy Conference
Heartening. That’s how I found last week’s PPMD PAAC Leadership and Advocacy Conference. Every time the whole community gets together, I leave feeling EMPOWERED. I like that word. Good word isn’t it? There is no limit to what I can do for my community. And I imagine I’m not the only…
ContinueAdded by Buddy Cassidy on February 27, 2017 at 4:00pm — No Comments
Staff Duchenne Research Update: February 2017
An Overview of Your Impact in Action
PPMD Grant Awards in the last Quarter of 2016 and first part of 2017.
PPMD Launches Long-Term…
ContinueAdded by Abby Bronson on February 24, 2017 at 1:29pm — No Comments
Staff PPMD Designates Ann & Robert H. Lurie Children's Hospital of Chicago a Certified Duchenne Care Center
Today Parent Project Muscular Dystrophy (PPMD) named Ann & Robert H. Lurie Children’s Hospital…
Added by PPMD on February 16, 2017 at 12:00pm — No Comments
Staff PPMD Recommends Marathon Convene Broad Array of Duchenne Stakeholders to Discuss Re-evaluation of Deflazacort Pricing
In light of the pause in commercialization of EMFLAZA™ (deflazacort)…
Added by PPMD on February 15, 2017 at 1:30pm — No Comments
Staff PPMD’s #EverySingleOne Tour: Los Angeles, CA
Children’s Hospital Los Angeles (CHLA) marked our first stop of the New Year on PPMD’s 2017 Every Single One Tour, held on January 21, 2017. We were thrilled to not only be holding another tour stop at one of …
ContinueAdded by PPMD on February 15, 2017 at 10:30am — No Comments
Staff Marathon Pharmaceuticals Pauses Commercialization of EMFLAZA™ (deflazacort)
UPDATE: February 13, 2017, 5:10 PM -- An additional statement from Marathon has been added to the blog
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Today, Marathon…
ContinueAdded by PPMD on February 13, 2017 at 4:00pm — No Comments
Staff Deflazacort Approved in the U.S. for Duchenne
UPDATE 2/10: Marathon has released links to new resources, including the EMFLAZA website (https://emflaza.com) and a Patient Support FAQ document (…
ContinueAdded by Pat Furlong on February 9, 2017 at 5:00pm — 2 Comments
Staff PPMD to Host Forum on Access to Emerging Therapies on February 13th, 2017
Following decades of strategic advocacy, research investment, and biopharmaceutical development, the U.S. Duchenne community recently arrived in the post-approval space and is poised to receive additional novel therapies from a robust innovation pipeline.…
Added by PPMD on February 7, 2017 at 1:57pm — No Comments
Staff PPMD’s Duchenne Drug Development Roundtable: Bringing Industry Together to Accelerate Research
Better. Faster. Now.
How do we apply the ‘regulatory learnings’ of the past 18 months back into our robust Duchenne pipeline to ensure as many effective therapies yield from our pipeline as possible?
Under the leadership of PPMD's Duchenne Drug Development Roundtable (DDDR), our community is determined to figure that out. Today is the first of a…
ContinueAdded by Abby Bronson on February 1, 2017 at 10:00am — No Comments
Featured Blog Posts
- PPMD Designates UC Davis a Certified Duchenne Care Center
- PPMD Working with FDA to Plan Landmark Pediatric Advisory Committee Meeting to Improve Clinical Trial Experience
- PPMD's Duchenne Drug Development Roundtable Meeting Series, Part 3: Clinical Trial Readiness from the Viewpoint of Clinicians and Infrastructure
- ACTION ALERT: Make Your Voice Heard on New Healthcare Proposal
- PPMD's Duchenne Drug Development Roundtable Meeting Series, Part 2: Optimizing Clinical Trials
- PPMD Designates Ann & Robert H. Lurie Children's Hospital of Chicago a Certified Duchenne Care Center
- Marathon Pharmaceuticals Pauses Commercialization of EMFLAZA™ (deflazacort)
Latest Blog Posts
- Celebrex is seen effective in DMD Mice
- Capricor Announces New Pre-Clinical Study Finds Repeat Doses of CAP-1002 Lead to Enhanced Exercise Capacity in Duchenne Muscular Dystrophy Disease Model
- PPMD & NIH to Host “Contractures in Duchenne and Other Neuromuscular Conditions”
- Solid Biosciences Provides Update On SGT-001 Clinical Development Program For Duchenne
- Pfizer Doses First Patient Using Investigational Mini-Dystrophin Gene Therapy for the Treatment of Duchenne Muscular Dystrophy
- 70 Members of Congress Send Letter of Strong Support for Duchenne Priorities for Next Year’s Budget
- Roche Restarts RG62026 Phase 2/3 Study
Most Popular Blog Posts
- April 25: FDA Advisory Committee Meeting for Eteplirsen - Live Webcast Information
- FDA Grants Accelerated Approval to First Drug for Duchenne Muscular Dystrophy
- September 28: FDA Advisory Committee Meeting for Ataluren - Live Webcast Information
- Changing the Paradigm: The Eteplirsen Ad Comm
- Deflazacort Approved in the U.S. for Duchenne
- A Historic Day for Duchenne
- Early morning calls
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