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All Blog Posts Tagged 'Staff' (327)


Staff
Prosensa Announces A Regulatory Path Forward for Drisapersen as a Potential Treatment for Duchenne

Prosensa Plans to Submit a New Drug Application to the FDA this Year; Dialogue with EMA continues with intent to seek approval

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…

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Added by PPMD on June 3, 2014 at 9:00am — No Comments


Staff
A Good Day for Duchenne

EMA grants Conditional Approval for Ataluren

 

Ataluren, PTC Therapeutics’ nonsense suppression drug received Conditional…

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Added by Pat Furlong on May 23, 2014 at 9:30am — 6 Comments


Staff
PTC Therapeutics Receives Positive Opinion from CHMP for Translarna (Ataluren)

Read the press release from PTC Therapeutics:



The first treatment for the underlying cause of Duchenne muscular dystrophy

SOUTH PLAINFIELD, NJ – May 23, 2014 – PTC Therapeutics, Inc.…

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Added by PPMD on May 23, 2014 at 9:30am — No Comments


Staff
Drisapersen Regulatory Discussions Ongoing & Re-Dosing on Track

Below you can find the latest update from Prosensa regarding a potential regulatory path forward for drisapersen.







Regulatory discussions ongoing & re-dosing on…

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Added by PPMD on May 22, 2014 at 8:00pm — No Comments


Staff
PPMD Congressional Champions Voice the Need for Action on the MD-CARE Act

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.…

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Added by Ryan Fischer on May 22, 2014 at 3:30pm — No Comments


Staff
Catena®/Raxone® Positive Results Reported from Phase III Duchenne Trial

Santhera reported positive results today from Catena®/Raxone® in their Phase III Duchenne trial. These results are supported by…

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Added by PPMD on May 22, 2014 at 1:00pm — No Comments


Staff
SMT C1100 Preliminary Results from Phase 1B Clinical Trial for Treatment of Duchenne

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…

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Added by PPMD on May 21, 2014 at 9:00am — No Comments


Staff
Halo Files "Complete Response" with FDA regarding Clinical Hold of HT-100 for Duchenne

Since being placed on clinical hold in December, Halo has generated additional data from the HT-100 clinical program and completed…

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Added by PPMD on May 13, 2014 at 3:00pm — 1 Comment


Staff
One More Reason to Register for PPMD’s 20th Connect Conference

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.

Not…

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Added by Kimberly Galberaith on May 13, 2014 at 1:30pm — No Comments


Staff
Unprecedented Collection of Duchenne Experts Developing Guidance for FDA

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…

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Added by Ryan Fischer on May 9, 2014 at 9:30am — No Comments


Staff
MD-CARE Act Results: Exon Skipping from Lab to Patient

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…

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Added by Ryan Fischer on May 8, 2014 at 4:30pm — No Comments


Staff
Results of PPMD-Funded Study Prompt Phase III Study of Tadalafil in Duchenne

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…

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Added by PPMD on May 7, 2014 at 4:00pm — No Comments


Staff
DuchenneConnect Annual Report: Registry Growth & Expanded Services

This past year has brought many new and exciting changes to DuchenneConnectthe 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…

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Added by Ann Martin on May 7, 2014 at 11:32am — No Comments


Staff
Prosensa Update on Drisapersen: Re-dosing plans are underway & 96-week data from an open-label extension study presented at the American Academy of Neurology (AAN)

Prosensa Provides Update on Drisapersen

Re-dosing plans are underway and 96-week data from an open-label extension study of drisapersen for the treatment of DMD presented at the American Academy of Neurology (AAN)…

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Added by PPMD on May 1, 2014 at 10:30am — 1 Comment


Staff
New Duchenne Therapies in Development

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…

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Added by Sharon Hesterlee on April 24, 2014 at 12:30pm — 3 Comments


Staff
Sarepta Therapeutics Announces Plans to Submit New Drug Application to FDA for Eteplirsen for the Treatment of Duchenne Muscular Dystrophy by Year End 2014

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!







Sarepta's Press…

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Added by PPMD on April 21, 2014 at 9:30am — 1 Comment


Staff
Success on Funding Letter, Time to re-engage on the MD-CARE Act



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…

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Added by Ryan Fischer on April 15, 2014 at 9:51am — No Comments


Staff
A New Care Guide to Give to Your Provider – Imperatives for Duchenne Care

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…

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Added by Kathi Kinnett on April 14, 2014 at 1:00pm — 9 Comments


Staff
PPMD Awards $100,000 in Exploratory Grants

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…

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Added by Sharon Hesterlee on March 25, 2014 at 11:42am — 2 Comments


Staff
Certified Duchenne Care Center Program

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…

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Added by Kathi Kinnett on March 20, 2014 at 9:00am — 2 Comments

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