May 2014 Blog Posts (15)

Congressional Committee That Funds the FDA Recognizes PPMD-Led Efforts on Benefit/Risk, Draft Guidance; Applauds Collaboration with Agency

For the first time ever, the appropriations bill that funds the Food and Drug Administration includes report language focused on Duchenne muscular dystrophy and championed by PPMD advocates.


This week the House Appropriations Committee approved the Fiscal Year 2015 Department of Agriculture and FDA spending bill, clearing the way for the full House to act on…


Added by Pat Furlong on May 30, 2014 at 11:22am — No Comments

A Good Day for Duchenne

EMA grants Conditional Approval for Ataluren


Ataluren, PTC Therapeutics’ nonsense suppression drug received Conditional Approval. This is a big moment for Duchenne, a…


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

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. (NASDAQ: PTCT) today announced that following its request…


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

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 track

Dear Patient Group…


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

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.…


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

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 additional respiratory function data. By including…


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

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 disease may be the cause.

The preliminary trial data will be reviewed…


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

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 several toxicology studies, and provided this information to…


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

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 anymore!



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

Certain Medicaid Waiver Payments May Be Excludable From Income

The Internal Revenue Service (IRS) recently reversed a policy and now allows parents of people with disabilities who receive Medicaid Home and Community Based waiver funds, for care giving services provided to their children, to exclude those funds from their incomes. Notice 2014-7 describes changes in the IRS policy. These changes may apply to foster and biological parents caring for a person with Duchenne muscular…


Added by Brian Denger on May 13, 2014 at 11:00am — No Comments

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 sponsors in the clinical development of medical products (i.e., human drugs and…


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

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…


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

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 circulation to normal levels. …


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

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…


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

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)

Read the press release.…


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

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