The Annual PPMD Connect Conferences have always been a haven for obtaining information around a broad range of topics applicable to the entire Duchenne community. Last year, we hosted a pre-conference meeting to address a single topic of need expressed by providers, parents, and patients alike – discrepancy in the care of people living with…Continue
Added by Kathi Kinnett on June 26, 2013 at 8:30am — No Comments
Today, I will be speaking with Treat NMD and European Medicines Agency (EMA) in response to their draft guidance on clinical studies in Duchenne muscular dystrophy. Elizabeth Vroom from Duchenne Parent Project in the Netherlands, Filippo Buccella from Parent Project Onlus in Italy, and I will be speaking on topics like clinical trials, inclusion criteria, and…Continue
The question of whether to use a cough assist or vest therapy for airway clearance in patients with Duchenne has come up several times. I connected with Drs. Richard Shell (Nationwide Children’s Hospital, Columbus, Ohio), Hemant Sawnani (Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio), and Jonathan Finder (Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania) for their expert opinions. Below is a summary of their thoughts.
“The Vest,” or “high…Continue
June 7/8, 2013
In February, 2013 EMA (Europeans Medicines Agency) released draft “Guidelines on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy” open for comment. This is the result of advances in basic and clinical research with an ever increasing number of clinical trials, recruiting small numbers of patients for these studies. This has raised a number of issues to include study design, the choice of appropriate…Continue
Coach To Cure, Sam's Day, North Texas FACES group, and now participating at PPMD’s Connect Conference at the end of June in Baltimore. These are just a few of the things 20-year-old Ben…Continue
Added by Will Nolan on June 14, 2013 at 12:39pm — No Comments
Earlier this week, PPMD hosted a webinar to discuss our FDA “aggressive collaboration” approach and the results from our recent benefit/risk survey. We conducted a rigorous survey of more than 100 parents whose children have Duchenne, gathering invaluable perspectives as to treatment priorities and risk threshold based on realistic but hypothetical treatments – information that PPMD is encouraging regulators to use to…Continue
Added by Holly Peay on June 14, 2013 at 10:00am — No Comments
With so many exciting happenings at PPMD and in our community, it is too easy to rush from one “fire” to the next and forget to celebrate our successes. Take a moment with us, and check out one success: our new Care for Duchenne pages.
We created the new…Continue
Results to Be Submitted to FDA with a Goal of Accelerating Review of Duchenne Therapies
PPMD has conducted a rigorous survey of more than 100 parents whose children have Duchenne, gathering invaluable perspectives as to treatment priorities and risk threshold based on realistic but hypothetical treatments - information that PPMD is encouraging regulators to use to accelerate review time and, ultimately, to speed access to…Continue
Added by PPMD on June 11, 2013 at 2:00pm — No Comments
My son Ryker is 14 months old. I just spoke with his neuromuscular specialist and she confirmed via his DNA and chromosome microarray that he has exon deletions 3-30 but the DNA test is showing that he is making minescule amounts of distrophin so she is cautiously diagnosing him with becker's but wants us to understand that by the time he is 5-7 it could change to Duchennes since he has such a large deletion and he could stop producing the small amount that he is now. She said that it is…Continue
Added by PPMD on June 7, 2013 at 8:30am — No Comments
For all the mothers, fathers and adolescents with Duchenne, I'd like to say this morning that Life is Good. Carlos graduated with the "Unsung Hero" and "Ethics" awards from his high school and perhaps more importantly, was asked to the senior prom. He and his date were the cutest couple on the dancefloor. There is so much to look forward to.
Added by Kathryn R. Wagner on June 6, 2013 at 7:54am — No Comments
Added by Ofir Arad on June 4, 2013 at 11:57pm — No Comments