April 2016 Blog Posts (15)
CRISPR/Cas9 Gene Editing
We finally have some very promising news for the development of adequate treatment for our boys with mutations requiring double or multi exon skipping!
In reading through this article about injecting a fully functional version of the dystrophin gene into the body using a harmless virus, it doesn't mention anything about applicability of multi-…
ContinueAdded by Jeffrey Kopp on April 27, 2016 at 3:37pm — No Comments
Staff Changing the Paradigm: The Eteplirsen Ad Comm
There is no way around it, today was hard. Hundreds of us – the largest gathering of Duchenne families in history – came together to tell the FDA that eteplirsen is safe, effective, and should be approved using the tools that…
ContinueAdded by Pat Furlong on April 25, 2016 at 10:30pm — 5 Comments
Staff April 25: FDA Advisory Committee Meeting for Eteplirsen - Live Webcast Information
On Monday, April 25, an FDA Advisory Committee will review eteplirsen for potential approval and use within our Duchenne community.
The Duchenne community is joining forces in a show of strength to demonstrate to the FDA our community’s support for our robust therapeutic…
ContinueAdded by PPMD on April 21, 2016 at 8:30am — No Comments
Staff Briefing Documents for Rescheduled Eteplirsen Ad Comm Released
The briefing documents for Sarepta’s Ad Comm on Monday, April 25th have been released. Like you, we…
ContinueAdded by PPMD on April 21, 2016 at 8:00am — No Comments
Staff PPMD Advocates Ensure Congress Recognizes Importance of Flexibility in FDA Reviews
All your calls, emails, and Hill visits during our February Advocacy Conference (in the pouring rain!) are yielding GROUND BREAKING RESULTS.
…
ContinueAdded by PPMD on April 19, 2016 at 4:00pm — No Comments
Staff PPMD Names Children’s Hospital Los Angeles 10th Certified Duchenne Care Center
PPMD Awards Tenth Clinic Certification as Part of Effort to Ensure People with Duchenne Receive Optimal Care
Children’s Hospital Los Angeles (CHLA) became the tenth clinic named a Certified Duchenne Care Center by Parent…
ContinueAdded by PPMD on April 19, 2016 at 9:00am — No Comments
Staff PPMD’s #EverySingleOne Tour Launched in Baltimore!
On Saturday, Duchenne community members from six states gathered together at the beautiful facilities at Kennedy Krieger in Baltimore – one of PPMD’s Certified Duchenne Care Centers – to hear…
ContinueAdded by Annie Kennedy on April 18, 2016 at 12:30pm — No Comments
Staff Senate Sends Strong Letter to FDA about Duchenne Therapies
Bipartisan Group of Senators Urge FDA to Utilize All Available Tools to Speed Access to Promising Duchenne Therapies
On Friday April…
ContinueAdded by Ryan Fischer on April 18, 2016 at 9:44am — No Comments
Staff Last Day for Early Bird Registration | PPMD's 2016 Connect Conference
Added by PPMD on April 15, 2016 at 10:30am — No Comments
Staff Parent Project Muscular Dystrophy Convenes Pulmonary Outcomes Workshop
Parent Project Muscular Dystrophy (PPMD) will convene a Pulmonary Outcomes Workshop in Bethesda, Maryland April 14-15. Leading physicians in…
Added by PPMD on April 14, 2016 at 2:26pm — No Comments
Staff PPMD’s Patient Preference Study about Pulmonary Outcomes – What we learned and why it matters
Late in 2015, Parent Project Muscular Dystrophy (PPMD) and our research partners at Johns Hopkins Bloomberg School of Public Health (JHSPH) released the results of…
ContinueAdded by PPMD on April 13, 2016 at 4:00pm — No Comments
Staff Catabasis Pharmaceuticals Initiates Part B of the MoveDMD Trial of CAT-1004 for Duchenne
Catabasis announced positive biomarker data from Part A of the MoveDMD trial, as well as initiation of dosing for the first patient for Part B (Phase 2) - a 12-week trial to assess the efficacy of CAT-1004 in Duchenne. We look forward to continued success from our partners at…
ContinueAdded by PPMD on April 12, 2016 at 9:30am — No Comments
Staff PPMD's 2016 Connect Conference Agenda Now Available!
PPMD is excited to unveil the agenda for the 22nd Annual Connect Conference in Orlando, June… |
Added by PPMD on April 11, 2016 at 1:16pm — No Comments
Staff ACTION ALERT: Please urge Your Senators to Sign Letter to FDA – Applying FDASIA Tools to Duchenne
Earlier this year, 109 members of the House of Representatives sent a powerful letter to the FDA urging them to “utilize all available tools, resources, and authorities” in reviewing a treatment for Duchenne muscular dystrophy. Congressional champions Congressmen Mike Fitzpatrick (R-PA), Bill…
ContinueAdded by Ryan Fischer on April 5, 2016 at 11:30am — No Comments
Staff How You Can Help with the Upcoming Ad Comm for Eteplirsen – Every Single Voice Matters!
We are just a few short weeks from the FDA Advisory Committee Meeting for eteplirsen on April 25th. PPMD is part of a united coalition of Duchenne organizations (MakeDuchenneHistory Coalition) working together to ensure we make the…
ContinueAdded by Ryan Fischer on April 4, 2016 at 10:30am — 1 Comment
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
Monthly Archives
2018
2017
- December (27)
- November (22)
- October (18)
- September (20)
- August (8)
- July (7)
- June (14)
- May (17)
- April (17)
- March (14)
- February (14)
- January (18)
2016
- December (13)
- November (10)
- October (26)
- September (15)
- August (16)
- July (10)
- June (13)
- May (12)
- April (17)
- March (18)
- February (20)
- January (23)
2015
- December (28)
- November (13)
- October (23)
- September (20)
- August (17)
- July (23)
- June (18)
- May (11)
- April (13)
- March (25)
- February (17)
- January (22)
2014
- December (18)
- November (13)
- October (18)
- September (16)
- August (10)
- July (21)
- June (18)
- May (18)
- April (50)
- March (32)
- February (10)
- January (12)
2013
- December (33)
- November (86)
- October (28)
- September (17)
- August (11)
- July (17)
- June (16)
- May (20)
- April (18)
- March (29)
- February (25)
- January (21)
2012
- December (14)
- November (21)
- October (35)
- September (31)
- August (28)
- July (19)
- June (16)
- May (26)
- April (24)
- March (33)
- February (26)
- January (18)
2011
- December (15)
- November (25)
- October (26)
- September (20)
- August (12)
- July (15)
- June (12)
- May (11)
- April (16)
- March (14)
- February (15)
- January (24)
2010
- December (24)
- November (11)
- October (11)
- September (11)
- August (20)
- July (15)
- June (14)
- May (19)
- April (15)
- March (20)
- February (17)
- January (27)
2009
- December (22)
- November (32)
- October (9)
- September (15)
- August (18)
- July (13)
- June (10)
- May (13)
- April (18)
- March (26)
- February (15)
- January (15)
2008
2007
© 2019 Created by PPMD.
Powered by
Badges | Report an Issue | Privacy Policy | Terms of Service
