GSK's Clinical Development Plan for Duchenne

GSK has released comprehensive details their four controlled clinical studies to evaluate the effect of the investigational medicine GSK2402968 (GSK’968), formerly known as PRO051, in boys with Duchenne, who have a dystrophin gene mutation amenable to an exon 51 skip.
January 26, 2012 | Read the update

Prosensa Raises €23 Million in New Equity Financing

Prosensa, the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet needs, announced today that it has raised €23 million in new equity financing. This new investment gives additional stability to the company and allows Prosensa to keep advancing the Duchenne programs as fast as possible.
January 25, 2012 | Read the release

Important Advocacy Webinar: PDUFA Reauthorization

PDUFA allows for the FDA to collects fees from drug manufacturers to, in turn, improve and expedite the approval process for drugs. The fees collected are used by the FDA to increase efficiency and timeliness with review of license application and increase their medical and scientific staff capability. Learn more about important legislation that affects the Duchenne community by joining us on this week's webinar (Wednesday, January 25th at 1pm Eastern).
January 23, 2012 | Learn more

PPMD Awards Stanley Froehner, PhD, $132,322 Grant

PPMD recently announced that it will award University of Washington’s Dr. Stanley Froehner a grant in the sum of $132,322 to continue his work with sildenafil (Viagra) and prednisone, as PDE5 inhibitors like sildenafil are brought to human clinical trials. According to Dr. Froehner’s research, studies of sildenafil, an FDA drug that amplifies the nNOS signaling pathway, demonstrate a marked improvement in heart and diaphragm function in the mdx mouse. In preparation for expanded clinical trials in Duchenne patients, his team will determine in mdx mice if sildenafil and prednisone have additive effects, or if prednisone can interfere with the positive effects of sildenafil. Since steroids are standard care in Duchenne, investigators want to understand how drugs will act when used in combination with steroids.
January 18, 2012 | Learn more about this project

NORD's letter to Congressman Stearns

The National Organization for Rare Disorders (NORD) believes that there are regulatory and legislative alternatives to the recent "Unlocking Lifesaving Treatments for Rare Diseases Act" (ULTRA) proposal that can more quickly advance the development of new drugs for rare diseases. 
January 16, 2012 | Read more

The January eNewsletter is here!

With the New Year, we are excited to bring you a revamped newsletter that gives you the inside scoop on the latest happenings in Duchenne research, advocacy, care, and the community. We will also bring you a new monthly feature, My Voice, featuring someone special living with Duchenne.
January 11, 2012 | Read more

Respecting Our Elders

PPMD Pres Pat Furlong & CO FACES Coordinator Ivy Scherbarth co-blog about learning from our patient community.

January 11, 2011 | Read More