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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…

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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…

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Added by PPMD on March 28, 2016 at 2:30pm — 1 Comment


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…

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Added 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…

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Added 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…

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Added 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 Alliance,…

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Added 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…

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Added 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…

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Added by PPMD on March 10, 2016 at 9:00am — No Comments


Staff
How You Can Participate in Upcoming FDA Advisory Committee Meetings

UPCOMING AD COMMS

Sarepta…

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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,…

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Added 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…

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Added 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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Added by PPMD on March 4, 2016 at 10:00am — 1 Comment


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…

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Added by PPMD on March 1, 2016 at 9:30am — No Comments

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