Today Parent Project Muscular Dystrophy (PPMD) named UC Davis Department of Physical Medicine and Rehabilitation Neuromuscular Disease Clinic (UC Davis) our 15th …Continue
Added by PPMD on April 6, 2017 at 12:30pm — No Comments
Later this spring – likely on or about May 18 – FDA’s Pediatric Advisory Committee will convene to consider the issue of allowing in-dwelling ports in Sarepta’s Essence trial.
Once again, our community is at a pivotal moment in our history where we can affect change and improve the clinical trial experience for our…Continue
Added by Annie Kennedy on April 5, 2017 at 10:00am — No Comments
Accelerating trials. Protecting our robust therapeutic pipeline. Ensure as many hits on goal as we can.
Priorities shared by all of us.
And to achieve this, PPMD has convened our industry partners through our Duchenne Drug Development Roundtable and held a series of small, targeted meetings this winter, each focused…Continue
Added by Annie Kennedy on April 5, 2017 at 11:00am — No Comments
“Who’s on first?”
Remember that classic Abbott & Costello routine? Well, trying to track the quickly-shifting healthcare bill has felt much like watching that routine. Complex, confusing, and exhausting. But the stakes here are no laughing matter.
We recognize that each member of our community views these…Continue
Added by Annie Kennedy on March 21, 2017 at 10:00am — No Comments
This week, PPMD convened Duchenne industry partners, experts in adaptive trial design and biostatistics, and innovative partners from outside of the Duchenne world who’ve revolutionized their clinical trial spaces for the second meeting of our Duchenne Drug Development Roundtable's 3-part meeting series, focusing on Clinical…Continue
Added by Abby Bronson on March 3, 2017 at 10:00am — No Comments
Today Parent Project Muscular Dystrophy (PPMD) named Ann & Robert H. Lurie Children’s Hospital of Chicago …
Added by PPMD on February 16, 2017 at 12:00pm — No Comments
UPDATE: February 13, 2017, 5:10 PM -- An additional statement from Marathon has been added to the blog
Today, Marathon Pharmaceuticals announced that they will pause the commercialization of…Continue
Added by PPMD on February 13, 2017 at 4:00pm — No Comments
UPDATE 2/10: Marathon has released links to new resources, including the EMFLAZA website (https://emflaza.com) and a Patient Support FAQ document (…Continue
Following decades of strategic advocacy, research investment, and biopharmaceutical development, the U.S. Duchenne community recently arrived in the post-approval space and is poised to receive additional novel therapies from a robust innovation pipeline.
Parent Project Muscular Dystrophy (PPMD) is deeply…Continue
Added by PPMD on February 7, 2017 at 1:57pm — No Comments
Added by Pat Furlong on January 30, 2017 at 10:00am — No Comments
Ensuring that the worlds of teens and adults with Duchenne 'grow bigger', as they grow older by expanding their reach – both literally and figuratively.
Combing your hair. Lifting a glass to your mouth. Washing your hands. Pressing elevator buttons. Zipping a coat. Opening a door. Turning on a light.…Continue
Today, PPMD announced a $2.2 million dollar grant to Dr. Jerry Mendell, Dr. Louise Rodino-Klapac (co-PI), and Nationwide Children’s Hospital. Our purpose in awarding this grant is to open and accelerate the field of gene therapy – a strategy that seemed impossible as a potential treatment for Duchenne as far back as the 1990s. If successful,…Continue
Added by Pat Furlong on January 6, 2017 at 9:00am — No Comments
Viral gastrointestinal (GI) viruses are no fun for anyone, but they are especially worrisome for a person living with Duchenne muscular dystrophy. GI viruses affect the GI track – the stomach and intestine (i.e., ‘gut’) – resulting in abdominal pain/discomfort, nausea, vomiting, intestinal pain/discomfort, cramps and diarrhea. Thank goodness they usually have…Continue
Added by Kathi Kinnett on December 21, 2017 at 10:30am — No Comments
We have so much momentum. Let’s seize this moment.
First of all, let me say THANK YOU! Your gift to PPMD on #GivingTuesday helped us surpass our goal, and we could not be more grateful. It is a wonderful way to kick off our holiday season—and we wanted to make sure you were among the…
Frequently, I get calls from mothers/sisters/aunts of people living with Duchenne about issues related to being a carrier of Duchenne:
In an effort to answer some of the questions that have arisen with the potential FDA approval of deflazacort in the U.S., PPMD has worked with both Masters and Marathon to put together the resource below, which we hope will provide clarity.
Added by Kathi Kinnett on October 31, 2016 at 2:00pm — No Comments
PPMD Awards Twelfth Clinic Certification as Part of Effort to Ensure People with Duchenne Receive Optimal Care
Added by PPMD on October 26, 2016 at 3:02pm — No Comments
It is hard to imagine anything worse than receiving the diagnosis of Duchenne. The dates and odysseys that brought us into this community differ, but the fervor we share to slow and stop the progression of this disease as it marches through our children is unparalleled in any community.
But today I know that there actually is something worse…Continue
Added by Pat Furlong on October 14, 2016 at 12:02pm — No Comments
Today, my sons are dancing! Today we ALL celebrate the first FDA approval in Duchenne. Today the FDA approved Exondys 51 (aka eteplirsen) injection, specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable…Continue
Added by Abby Bronson on September 8, 2016 at 9:43am — No Comments