Parent Project Muscular Dystrophy

AVI BioPharma to Present Company Overview at the 14th Annual BIO CEO & Investor Conference

Thu, 02 Feb 2012 17:00:08 -0500

AVI BioPharma, a developer of RNA-based therapeutics, announced today that Chris Garabedian, AVI's president and CEO, will present a company overview at 9:30 a.m. EST on Tuesday, Feb. 14, at the 14th Annual BIO CEO & Investor Conference at the Waldorf Astoria in New York City. The presentation will be webcast live under the events section of AVI's website at www.avibio.com and will be archived there following the presentation for 90 days.

Despite Flat Budget, NIH’s TRND Program Tries to Spark Interest in Rare and Neglected Diseases

Thu, 02 Feb 2012 16:59:50 -0500

With 14 approved programs in total, TRND has progressed two into clinical development. For the past two years NIH has been trying to double its funds for developing orphan drugs. But the FY 2012 budget offered more of the same, as in the same $24 million of annual funding set aside for NIH’s Therapeutics for Rare and Neglected Diseases (TRND) program since its creation in 2009. The institute had sought $50 million for TRND this year and last.

Important Advocacy Webinar: PDUFA Reauthorization

Tue, 31 Jan 2012 11:52:06 -0500

As PPMD heads into another year advocating in Washington, we encourage all members of our community to learn more about drug development and the FDA.

GSK's Clinical Development Plan for Duchenne

Thu, 26 Jan 2012 12:44:01 -0500

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.

Prosensa Raises €23 Million in New Equity Financing

Thu, 26 Jan 2012 12:43:44 -0500

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.

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

Wed, 18 Jan 2012 16:01:10 -0500

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.

NORD's letter to Congressman Stearns

Mon, 16 Jan 2012 16:27:15 -0500

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.

The January eNewsletter is here!

Mon, 16 Jan 2012 16:26:59 -0500

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.

Enzyme function may discover muscular dystrophy therapies

Mon, 16 Jan 2012 16:26:46 -0500

The function of the glycosylating enzyme involved in muscular dystrophy, brain development and infection by arenaviruses could reveal new therapies. Researchers at the University of Iowa have mapped the function of the enzyme LARGE and believe that the knowledge could help to screen potential muscular dystrophy therapies. The LARGE enzyme is known to add a critical sugar chain onto an important membrane protein called dystroglycan.

Make 2012 the year you Conquer the Canyon to End Duchenne!

Wed, 28 Dec 2011 12:57:11 -0500

PPMD and Conquer The Canyon have joined forces to bring you Conquer The Canyon to End Duchenne, a once in a lifetime, 3-night/4-day walking, hiking and active adventure at one of the "Grandest" natural wonders of the world - The Grand Canyon! From April 20-23, 2012 PPMD is inviting you to join us on this exclusive, once in a lifetime adventure to help us end Duchenne.

Important Webinar: Understanding Biglycan

Wed, 28 Dec 2011 12:56:45 -0500

Dr. Justin Fallon, our December Person of the Month, presented an update on biglycan at this month's webinar on December 20. Dr. Fallon discussed the history of biglycan as a potential therapy for Duchenne, and outlined next steps for he and his team.

Summit Secures $1.5M Agreement with Duchenne Organizations

Mon, 19 Dec 2011 17:10:19 -0500

PPMD is happy to be supporting Summit PLC with its utrophin upregulation drug candidate SMT C1100. The funding provided will enable Summit to manufacture a new formulation of SMT C1100 and conduct a Phase I clinical trial in healthy volunteers.

Important Webinar: Understanding Biglycan

Mon, 19 Dec 2011 17:10:01 -0500

Dr. Justin Fallon, our December Person of the Month, will be presenting an update on biglycan at this month's webinar on December 20, 1pm eastern. Dr. Fallon will briefly discuss the history of biglycan as a potential therapy for Duchenne, and outline next steps for he and his team.

PPMD Supports Therapeutic Credit

Mon, 19 Dec 2011 17:09:45 -0500

PPMD joined a list of important organizations in the patient and health advocacy community as part of a letter urging Congress to support an extension of the Therapeutic Discovery Project Tax Credit. This tax credit encourages investment in new promising therapies to prevent, diagnose, and treat acute and chronic diseases. PPMD signed onto the original request in 2009 supporting the initiative then. We are hopeful the extension is enacted.

For Academics - NeuroNEXT Webinar

Wed, 14 Dec 2011 16:39:40 -0500

NINDS is hosting a webinar on the new clinical trial network for neurological disorders. NeuroNEXT provides trial site and coordinating center infrastructure to facilitate rapid development and implementation of protocols in neurological disorders. Information on accessing the webinar is linked below. You are encouraged to participate to learn about the NeuroNEXT network. Please pass this on to anyone that might be interested.

December eNewsletter is Here!

Thu, 08 Dec 2011 10:44:30 -0500

In this month’s newsletter we are pleased to announce three conferences in 2012, each promising to give attendees a unique and fulfilling experience. Plus, meet our Person of the Month and check out some important new downloads.

Let's make it happen

Tue, 06 Dec 2011 10:56:58 -0500

PPMD's yearly Holiday Appeal directs all monies to a specific research program. This year, PPMD has chosen to further fund Dr. Justin Fallon and Tivorsan Pharmaceuticals’ research in biglycan. Biglycan shows promise for all our sons. It potentially can benefit all mutations of Duchenne and Becker patients. Dr. Fallon and Tivorsan now see an opportunity to move this research quickly to a clinical trial. We are asking for your help to make it happen.

Ataluren Update

Mon, 12 Dec 2011 10:57:42 -0500

PTC has announced a study that will be open to all patients, regardless of ambulation status, who received ataluren in a prior PTC-sponsored DBMD study in Europe, Israel or Australia. This clinical study will have an open-label design, similar to the ongoing clinical trial in the US for previous ataluren trial patients.

Webinar: Fat Embolism Syndrome Following Mild Trauma in Duchenne

Tue, 29 Nov 2011 10:58:24 -0500

With growing concern in the Duchenne community regarding Fat Embolism Syndrone (FES) after a child falls or sustains what would normally be considered a minor injury, Parent Project Muscular Dystrophy has asked a leading expert to present an important webinar on FES.

Dr. Laura McAdam from Holland Bloorview Kids Rehabilitation Hospital in Toronto, Ontario will be discussing what is FES, what are the symptoms, and what tests can be done to help facilitate the diagnosis. They will also discuss other potential diagnoses that can present this way, as well as the supportive treatment when FES is diagnosed.

The ultimate goal of this webinar is to empower you with knowledge to have discussions with emergency room doctors and increase the possibility of an FES diagnosis at a critical time.

Letter to The U.S. Department of Health and Human Services (HHS)

Tue, 29 Nov 2011 10:59:08 -0500

Over the summer, The U.S. Department of Health and Human Services (HHS) announced that the federal government is contemplating various ways of enhancing the regulations overseeing research on human subjects. PPMD was thrilled to hear that before changes were made to the regulations, the government was requesting input from the public. PPMD felt this to be an important opportunity to weigh in on how this affects the Duchenne community and what suggestions can be made to better meet the unique needs of our community.