March 2016 Blog Posts (13)
Staff Summit Reports Positive Interim Data from New Formulation of SMT C1100 in Duchenne
Summit Therapeutics announced positive interim data from a Phase 1 clinical trial evaluating a new oral formulation of its lead utrophin modulator, SMT…
Added by PPMD on March 30, 2016 at 9:30am — No Comments
Staff ReveraGen BioPharma Receives $3M for Phase 2 Studies of Vamorolone in Duchenne
PPMD is thrilled to see the continued progress of VBP-15, now known as Vamorolone. Progress to date includes a first in-human study of healthy volunteers and animal data which suggests it has none of the side effects…
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Staff Update on Gene Replacement/Repair Strategies for Duchenne Muscular Dystrophy
There has been a flurry of press recently on gene therapy in Duchenne muscular dystrophy and CRISPR technology. With this barrage of information, PPMD wanted to take a deeper dive into gene therapy as a potential treatment for Duchenne.
An Expert…
ContinueAdded by Abby Bronson on March 25, 2016 at 9:30am — No Comments
Staff PPMD is Initial Partner in OnPAR — Online Partnership to Accelerate Research
The National Institutes of Health (NIH) is one of the biggest funders of medical research in the US, and projects it has funded have touched almost every Duchenne therapeutic in development now in some way. In FY2016, the NIH…
ContinueAdded by Abby Bronson on March 24, 2016 at 9:30am — No Comments
Staff DuchenneConnect & UCLA Recruiting Patients for Genetic Modifiers Study
We know that changes or mutations in the Dystrophin gene are what cause Duchenne and Becker but we don’t have a good understanding of why people within the same family with the same genetic change…
ContinueAdded by Ann Martin on March 15, 2016 at 10:49am — No Comments
Staff How You Can Participate in the Eteplirsen Ad Comm
The Duchenne community is working closely together to prepare for the April 25 Advisory Committee Meeting for Sarepta’s therapy eteplirsen. PPMD, the Duchenne…
ContinueAdded by PPMD on March 15, 2016 at 9:00am — No Comments
Staff The Importance of Every Single Breath—PPMD to Host Pulmonary Outcomes Meeting
We are well aware that changes in pulmonary function occur as Duchenne progresses. In Duchenne, respiratory muscles (including the diaphragm) weaken and it becomes more difficult to take full…
ContinueAdded by Kathi Kinnett on March 10, 2016 at 11:30am — No Comments
Staff FDA Releases New Date for Eteplirsen Ad Comm
At long last we have received information regarding the rescheduled Advisory Committee for Sarepta (eteplirsen). The Ad Comm will take place on Monday, April 25 at the College…
ContinueAdded by PPMD on March 10, 2016 at 9:00am — No Comments
Staff How You Can Participate in Upcoming FDA Advisory Committee Meetings
Added by PPMD on March 10, 2016 at 9:00am — No Comments
Staff A Win in Washington Today – For Every Single Person Living with Duchenne
Our Duchenne community is on our way to achieving ANOTHER milestone.
MD-CARE Act passed 2001
MD-CARE Act reauthorized 2008
MD-CARE Act Amended 2014
Heavy Engagement within PDUFA V; FDASIA reauthorization 2012
Today,…
ContinueAdded by Annie Kennedy on March 9, 2016 at 11:30am — No Comments
Staff Gene Therapy Today: Notes from PPMD’s Duchenne Gene Therapy Forum
Gene therapy.
Those two words have been tossed around for some time, starting in the late 1990s when several gene therapy trials started, but ended with fatal results. Now, when the field has learned a lot from those initial results and with optimized technologies, we come back to gene…
ContinueAdded by Abby Bronson on March 8, 2016 at 12:00pm — 4 Comments
Staff PPMD Launches the Every Single [One] Tour
For 22 years, PPMD has brought together families with leading experts, care providers, and industry partners through our Annual Connect Conference,…
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Staff Registration for PPMD's 2016 Connect Conference is Now Open
Parent Project Muscular Dystrophy's Annual Connect Conference is a unique convergence of industry partners, scientific leaders, medical providers, people living…
ContinueAdded by PPMD on March 1, 2016 at 9:30am — 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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