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All Blog Posts Tagged 'Staff' (271)


Staff
How to Decode Duchenne

We are excited to announce that Parent Project Muscular Dystrophy and Sarepta Therapeutics are joining forces on a genetic testing program for patients with Duchenne or Becker…

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Added by Pat Furlong on October 17, 2013 at 9:00am — No Comments


Staff
My Voice: Jake Pritchard, 9 years old

When a family hosts a fundraising event for Duchenne, we are thrilled and extremely grateful. When a family hosts the same event six years in a row, we are overwhelmed! This year the Pritchard family, along with their friends – Meghan and Jason Alpern – in Matthews, North Carolina will host the…

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Added by Will Nolan on October 15, 2013 at 11:30am — No Comments


Staff
Pushing the FDA to Accelerate Treatments

We all know it: rare diseases, especially progressive pediatric disorders like Duchenne, are different than common diseases. The FDA knows it too, but they don’t have a plan for how to treat them differently. They are working on different ways to weigh the benefits and risks of treatments for…

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Added by Holly Peay on October 10, 2013 at 9:30am — No Comments


Staff
Clinical Trial Expectations

I was privileged to share some of the data PPMD has collected in our Clinical Trial Expectations study at the World Muscle Society poster session. Many clinicians and industry and academic scientists came to the poster to learn about our community thoughts and preferences, and were quite interested in how they might…

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Added by Holly Peay on October 7, 2013 at 10:58am — No Comments


Staff
World Muscle Society Congress: Considering Care

Care related research was popular at this year’s 18th International World Muscle Society Congress. While this is not a complete summary, I will try to hit the highlights. 

 

Several poster sessions, as well as the first portion of the Sarepta Symposium,…

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Added by Kathi Kinnett on October 4, 2013 at 12:59pm — No Comments


Staff
World Muscle Society Congress: Next Generation Exon-Skipping & Clinical Trial Updates

The World Muscle Society Meeting in Monterey, California saw reports on a number of topics of interest to the community. On Thursday poster sessions covered advances in the next generation of exon-skipping and updates on various clinical trials.

Next Generation Exon-Skipping…

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Added by Sharon Hesterlee on October 4, 2013 at 9:30am — No Comments


Staff
Impact of Drisapersen Phase 3 Data on Prosensa Programs

Prosensa has provided the following update regarding the impact of the drisapersen phase 3 data on its programs: 

While we are disappointed and surprised with the unexpected outcome of the drisapersen phase 3 study, we remain very committed to drisapersen, the Duchenne program at…

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Added by PPMD on September 24, 2013 at 11:30am — No Comments


Staff
GSK and Prosensa announce primary endpoint not met in Phase III study of drisapersen

UPDATE AS OF 10/4/13: We received the following Q&A from GSK today, addressing many of the community's questions. Click here.

Today we learned that GSK’s phase III study of the exon 51 skipping drug drisapersen failed to show a statistically significant improvement in the…

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Added by Sharon Hesterlee on September 20, 2013 at 11:00am — 14 Comments


Staff
PPMD Leadership on Patient-Focused Drug Development Nets Invitation to Present at Two Influential Meetings

Parent Project Muscular Dystrophy (PPMD) has been invited to present at two upcoming stakeholder meetings.



The invitations, to PPMD Founding President & CEO Pat Furlong, were extended by leaders in the U.S. government in recognition of PPMD's groundbreaking work to advance the patient and parent perspective in the drug development and review…

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Added by PPMD on September 10, 2013 at 9:44am — No Comments


Staff
Spiritual Journey to the Arches

Guest post by Jill Anne Castle, mom to Oliver, 11, and Anthony, 13 and living with Duchenne. Jill is an Educational Consultant and PPMD FACES of Arizona Volunteer Coordinator.…



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Added by PPMD on September 9, 2013 at 8:30am — 2 Comments


Staff
How I Wish

Guest post by Ivy Scherbarth. Ivy is a Colorado/Wyoming FACES Coordinator for PPMD and mom to Hazel, age 8, and Rain, age 6. Rain has Duchenne muscular dystrophy. Follow Ivy at her blog,…

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Added by PPMD on September 6, 2013 at 12:46am — 2 Comments


Staff
2013 MDCC Meeting

The Muscular Dystrophy Coordinating Committee (MDCC) had its 10th meeting on August 26, 2013 at the National Institutes of Health offices in Bethesda, MD.  The members of the MDCC come from federal agencies involved in one or more muscular dystrophy programs as well as public representatives from several organizations representing one or more forms of muscular dystrophy.  There are representatives from…

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Added by Brian Denger on September 4, 2013 at 3:00pm — No Comments


Staff
Back to School - Duchenne Resources

It’s that time of year again – the start of another school year. While we put our faith in schools to provide a proper, inclusive education for our children, faith is not all we have in our arsenal for ensuring this is the case for students living with Duchenne.

 

As parents, you are…

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Added by Ryan Fischer on September 3, 2013 at 10:45am — No Comments


Staff
My Voice: Mitchell Peterson, 17 years old

Saturday, August 17 marks the 15th Annual Mitchell's Run Thru Rockford. And for 15 years, the Peterson family has been raising money and awareness for Duchenne. Mitchell Peterson, 17 years old, has been the inspiration behind this event that continues to grow in numbers and momentum. He refuses to be defined by Duchenne and as he begins his senior year of high school, is already planning for a career after college. The Peterson…

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Added by Will Nolan on August 15, 2013 at 12:29pm — No Comments


Staff
Major Changes in Physical Therapy Coverage for Individuals with Duchenne and Becker

Obtaining physical therapy (PT) services for individuals with Duchenne and Becker muscular dystrophy, and other conditions for which PT does not result in improvements in range of motion or function, has been a huge source of frustration for patients, parents, and providers.

A recent…

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Added by Kathi Kinnett on August 15, 2013 at 8:00am — No Comments


Staff
PPMD to Fund $1 Million in Exon Skipping Projects

PPMD announced today that it will fund up to $1 million in projects by mid-2014 focused on the development of antisense oligonucleotides or other techniques to skip exons that are not currently in clinical development, or for multi-exon skipping projects, including the skipping of…

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Added by PPMD on August 14, 2013 at 11:00am — 1 Comment


Staff
Salt, Steroids, and Duchenne

“Hypertension” is the fancy word for high blood pressure. Blood pressure is the measure of how much pressure it takes the left ventricle of the heart to squeeze the blood out (the top number, or systolic pressure) and the pressure inside the left ventricle of the heart when the heart is relaxed (the…

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Added by Kathi Kinnett on August 14, 2013 at 10:30am — 3 Comments


Staff
MD-CARE Act: Is your member in a target state or district?

UPDATED 2/11/14

As we move into the start of 2014 and the February Advocacy Conference we are hopeful to gain momentum to move MD-CARE Act to committee action this year. With 78 cosponsors in the House and 17 in the Senate, we need your help to up our…

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Added by Ryan Fischer on July 31, 2013 at 9:30am — No Comments


Staff
Sarepta Therapeutics announces plans to submit New Drug Application to FDA for Eteplirsen – A significant step for the Duchenne community

Today is a landmark day for the entire Duchenne community. Not too long ago, the Duchenne therapy landscape was a barren field with little to no life. Thanks to a commitment to our patients and the field by the U.S. government, international governments, the venture community and industry, this landscape has changed markedly to the point where today we have the announcement for a…

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Added by Pat Furlong on July 24, 2013 at 11:00am — 1 Comment


Staff
Advocating for Open Access

In 2008, PPMD joined the Genetic Alliance and others in the rare disease community, in support of “open access” – essentially ensuring that all NIH funded researchers make available to the public published peer-reviewed manuscripts and articles on a searchable website like PubMed Central or the National Library of Medicines. Making them freely accessible to the public within 12 months of publication.

 …

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Added by Ryan Fischer on July 18, 2013 at 4:30pm — No Comments

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