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All Blog Posts Tagged 'Staff' (332)


Staff
Healthy Summer Snacking & Activity



It’s summer – finally! Growing children are hungry children, and hungry children don’t usually go for the healthy snacks first.



As parents, we try everything we can to get them to eat right: reasoning (this sometimes works), bribery (generally referred to as “creative parenting”) and…

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Added by Kathi Kinnett on June 16, 2014 at 12:30am — No Comments


Staff
"Right to Try" State Model Legislation

The Goldwater Institute of Arizona has designed state model legislation, known as a "Right to Try" bill, which would make experimental drugs available to terminally-ill patients without the FDA's approval. Under this legislation, a patient with "an advanced stage of a disease with an unfavorable prognosis and no known…

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Added by PPMD on June 16, 2014 at 12:00am — 2 Comments


Staff
Clinical Development of HT-100 for Duchenne Has Resumed

Phase 1b/2a Clinical Program Resumes Enrollment and Dosing in DMD Patients 

Preliminary Clinical Data to be presented at Upcoming Medical Conference 

Read the news release from DART Therapeutics:

DART Therapeutics Continues Clinical Development of HT-100 for Duchenne Muscular Dystrophy 

Cambridge, Mass.—June 12,…

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Added by PPMD on June 12, 2014 at 12:23pm — 5 Comments


Staff
PPMD Benefit/Risk Study Results Published in Clinical Therapeutics

PPMD is thrilled that results from the recent benefit/risk project our team led have been published in Clinical Therapeutics. The article concludes that caregivers are willing to accept a…

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Added by Holly Peay on June 9, 2014 at 1:30pm — No Comments


Staff
Lend Your Voice to the FDA Draft Guidance on Duchenne to Speed the Development of Therapies

Strength Happens Together

For the past four months, over 80 experts in Duchenne (including patient representatives and groups, industry, clinicians, and researchers) have been working…

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Added by Ryan Fischer on June 4, 2014 at 4:00pm — No Comments


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

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