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…

EMA grants Conditional Approval for Ataluren

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

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 for…

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.…

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 pulmonary…

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…

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!

Buckle…

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…

The process for writing a Draft Guidance on Duchenne for the FDA and industry is in full swing.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…

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…

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. …

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…

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.…