Summit Therapeutics announced positive interim data from a Phase 1 clinical trial evaluating a new oral formulation of its lead utrophin modulator, SMT C1100, for Duchenne. In this dose escalating clinical trial's…
Added by PPMD on March 30, 2016 at 9:30am — No Comments
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 of steroids, does not impact linear growth, and does not promote…
ContinueThere 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 Opinion…
ContinueAdded by Abby Bronson on March 25, 2016 at 9:30am — No Comments
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 is investing nearly $32.3 billion in medical research, and of…
ContinueAdded by Abby Bronson on March 24, 2016 at 9:30am — No Comments
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 (genotype) can be so different in their symptoms (phenotype). The…
ContinueAdded by Ann Martin on March 15, 2016 at 10:49am — No Comments
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, the Center for DMD at UCLA, Jett Foundation, and the Race to Yes…
ContinueAdded by PPMD on March 15, 2016 at 9:00am — No Comments
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 breaths in, fully expand the lungs, hold full breaths, breath…
ContinueAdded by Kathi Kinnett on March 10, 2016 at 11:30am — No Comments
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 Park Marriott Hotel & Conference Center (a non-FDA…
ContinueAdded by PPMD on March 10, 2016 at 9:00am — No Comments
Added by PPMD on March 10, 2016 at 9:00am — No Comments
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, March 9, 2016 on our way to another banner day with the…
ContinueAdded by Annie Kennedy on March 9, 2016 at 11:30am — No Comments
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 therapy as a viable option for genetic diseases. This renewed…
ContinueAdded by Abby Bronson on March 8, 2016 at 12:00pm — 4 Comments
For 22 years, PPMD has brought together families with leading experts, care providers, and industry partners through our Annual Connect Conference, FACES…
ContinueParent Project Muscular Dystrophy's Annual Connect Conference is a unique convergence of industry partners, scientific leaders, medical providers, people living with Duchenne, and their families. This exceptional meeting has grown to…
ContinueAdded by PPMD on March 1, 2016 at 9:30am — No Comments
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