More good news from the FDA! This news from Prosensa continues to demonstrate that FDA recognizes the urgent needs of this community, the importance of…Continue
Added by PPMD on June 3, 2014 at 9:00am — No Comments
EMA grants Conditional Approval for Ataluren
Ataluren, PTC Therapeutics’ nonsense suppression drug received Conditional…Continue
The first treatment for the underlying cause of Duchenne muscular dystrophy
SOUTH PLAINFIELD, NJ – May 23, 2014 – PTC Therapeutics, Inc.…
Added by PPMD on May 23, 2014 at 9:30am — No Comments
Added by PPMD on May 22, 2014 at 8:00pm — No Comments
On May 20th, the House Energy and Commerce Subcommittee on Health held a hearing entitled, 21st Century Cures: The President’s Council of Advisors on Science and Technology (PCAST) Report on Drug Innovation.
The hearing was led by Chairman Joe Pitts (R-PA) and is part of the new 21st Century Cures initiative that seeks to accelerate the discovery, development and delivery of promising new treatments to patients.…Continue
Added by Ryan Fischer on May 22, 2014 at 3:30pm — No Comments
Santhera reported positive results today from Catena®/Raxone® in their Phase III Duchenne trial. These results are supported by…Continue
Added by PPMD on May 22, 2014 at 1:00pm — No Comments
In the first study of a utrophin up-regulating drug in Duchenne, British biotech company Summit PLC reports that its drug SMT C1100 showed a good safety profile in twelve boys with Duchenne. Plasma levels of the drug were variable and the company speculates that diet and the course of the…Continue
Added by PPMD on May 21, 2014 at 9:00am — No Comments
Since being placed on clinical hold in December, Halo has generated additional data from the HT-100 clinical program and completed…
Every year on the Saturday night of the Connect Conference, PPMD hosts a final dinner. Usually there is a speaker or two, dinner and drinks and a last moment to talk with friends.
Added by Kimberly Galberaith on May 13, 2014 at 1:30pm — No Comments
The process for writing a Draft Guidance on Duchenne for the FDA and industry is in full swing.
As you know, the purpose of this guidance is to assist…Continue
Added by Ryan Fischer on May 9, 2014 at 9:30am — No Comments
Originally signed into law in 2001, the MD-CARE Act has birthed key discoveries in the muscular dystrophies, including the scientific justification for why exon skipping therapies could potentially work in humans.
Without it being…Continue
Added by Ryan Fischer on May 8, 2014 at 4:30pm — No Comments
A recent PPMD-funded study at Cedars Sinai Medical Center in Los Angeles (published in the May 7, 2014, online issue of Neurology®) demonstrates that those with Duchenne have impaired circulation in their muscles, which may contribute to muscle weakness, and that the approved vasodilator tadalafil can restore that…Continue
Added by PPMD on May 7, 2014 at 4:00pm — No Comments
This past year has brought many new and exciting changes to DuchenneConnect, the robust and cutting-edge registry and resource that connects Duchenne and Becker patients with actively recruiting clinical trials and research studies, and educates patients and families about Duchenne and Becker research. The registry, which was established in 2007, has matured beyond a standard registry and into an innovative…Continue
Added by Ann Martin on May 7, 2014 at 11:32am — No Comments
As we finalize the agenda for this year’s Connect Conference, I am once again amazed at how much of a challenge it is to squeeze in all of the talks on different therapeutic approaches to treating Duchenne—in fact, there are too many to cover comprehensively this year. Sometimes it’s good to remember that mutation-specific approaches like exon skipping, although promising and…Continue
Great investor call from Sarepta this morning on the heels of the news about positive signals from the FDA for submitting a new drug application! We are speaking with the company this morning and will release a detailed statement this afternoon. Congratulations to everyone!
Thanks to your efforts, we closed the House and Senate funding letters with the support of 30 House members and 12 Senators. Great work! We appreciate everyone who sent emails and made calls to their members of Congress.
The members below…Continue
Added by Ryan Fischer on April 15, 2014 at 9:51am — No Comments
Unfortunately, the care of people living with Duchenne muscular dystrophy differs from country to country, from state to state, and even from center to center. Email and stories that we get daily confirm that, despite the availability of Care Standards there is still significant practice variability, including centers and locations where clinicians…Continue
PPMD is thrilled to award two exploratory grants as part of our ongoing grant program. Peter Arthur of the University of Western Australia was awarded $50,000 to finish testing a molecule called procysteine in animal models in preparation for a human clinical trial. Procysteine is an antioxidant that has…Continue
Right now, there are more than 10-12,000 young people diagnosed and living in the United States with Duchenne. These young people are being cared for by clinics both near and far. Every one of them deserves the best care and treatment possible. Families want care and treatment to be transparent; they want to know who is delivering care to their child, what comprehensive care looks like, and which centers are delivering that care. For this reason, a group of experts agreed to give families…Continue