Santhera Pharmaceuticals announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion for its Type II extension application for Raxone® (idebenone) in Duchenne. Santhera plans to appeal the opinion and will seek a re-examination by the CHMP. We appreciate…Continue
Added by PPMD on September 15, 2017 at 8:00am — No Comments
Today Parent Project Muscular Dystrophy (PPMD) named Nemours/Alfred I duPont Hospital for Children (Nemours) in Wilmington, Delaware our 17th Certified Duchenne Care Center.
PPMD’s Certified Duchenne Care Center Program,…Continue
Added by PPMD on September 14, 2017 at 8:30pm — No Comments
There are some days when I feel like our community is in its infancy. When I look at where we are in terms of access, therapeutic options, combinations, care implementation…
But more often than not, I am overwhelmed with gratitude for the infrastructure and resources…
Added by Annie Kennedy on September 13, 2017 at 5:00pm — No Comments
We kicked off our fall Every Single One Tour stops in West Virginia this past weekend! We appreciate all of the families, speakers, and volunteers that spent the day with us in…Continue
Added by Kathi Kinnett on September 13, 2017 at 4:30pm — No Comments
Paperwork – oh the paperwork!
Each time you visit a physician’s office or hospital, we leave with piles of forms and papers detailing the visit. The providers seen, the prescriptions written, results from tests conducted, instructions for follow up, and more. And each of those pieces of…Continue
Added by Annie Kennedy on September 13, 2017 at 10:00am — No Comments
The deadline to submit a written comment for the FDA Advisory Committee Meeting on ataluren is this upcoming Thursday, September 14th.
To make a…Continue
Added by PPMD on September 12, 2017 at 9:30am — No Comments
The dynamics of weight loss are intriguing, in take a look at the many because they came from desire to shed some weight in comparison to the small number who do. You would think with all the diet plans and dietary supplements, that weight problems would go away. But that hasn't came about, so something must be wrong. The sheer number of programs and products out there helps it to be difficult to find out what is effective and what is not. And it is far from as if one solution is effective…Continue
Added by Louie Lewis on September 11, 2017 at 9:56am — No Comments
PPMD is excited to announce next year’s lineup of areas we will visit as part of our End Duchenne Tour!
Combining each of the pillars that make up PPMD’s mission, the End Duchenne Tour brings updates on research, advocacy, and care to cities across the…
Added by PPMD on September 8, 2017 at 5:36pm — No Comments
Added by PPMD on September 6, 2017 at 10:00am — No Comments
Earlier this year, Bristol-Myers Squibb (BMS) announced it had entered into an agreement to license BMS-986089 (RG6206), an anti-myostatin adnectin in development for Duchenne muscular dystrophy, to Roche. Today, the Roche team has…Continue
Added by PPMD on September 5, 2017 at 9:30am — No Comments
Yesterday, Kymriah, a gene therapy made by Novartis that treats an aggressive type of leukemia was approved for children and young adults. The new commissioner of the FDA, Dr. Scott Gottlieb,…Continue
Added by Abby Bronson on August 31, 2017 at 1:00pm — No Comments
Added by Kathi Kinnett on August 28, 2017 at 11:30am — No Comments
With trials in gene therapy on the horizon, and terms like "cassette", "construct", "promoter", and "transduce" being thrown around, we want to ensure that you, our Duchenne Community, has all the knowledge you need to navigate the world of gene therapy.
Added by Abby Bronson on August 23, 2017 at 8:00pm — No Comments
The Duchenne community is now preparing for the September 28 Advisory Committee Meeting for PTC’s therapy ataluren. Whether submitting written…Continue
Added by PPMD on August 22, 2017 at 11:00am — No Comments
May 12-13, 2016, PPMD convened a workshop of 48 opinion leaders, research experts and clinicians from the US and Canada, on the subject of Duchenne and bone biology. Bone health has long been a…Continue
Added by Kathi Kinnett on August 14, 2017 at 10:00pm — No Comments
Hope is critical to survival. Hope helps us get through those times of darkness with the thought that there is something new on the horizon, a potential new destination. Hope enables us to focus on meeting challenges even in the midst of adversity. Hope allows us to see that there might be…Continue
Added by Ann Lucas on August 7, 2017 at 2:00pm — No Comments
The PDUFA VI bill – known as FDA Reauthorization Act in the House (FDARA) – passed the House of Representatives early in July with strong bi-partisan support. It then moved to the Senate but consideration was delayed until after the Senate healthcare reform deliberations were completed. Today, though, we are excited that a companion bill (mirroring the House version) passed in the Senate! It will now move to the President’s desk where it is expected…Continue
Added by Annie Kennedy on August 3, 2017 at 8:30pm — No Comments
Today – the Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act, S. 1052 – led by Sen. Roger Wicker (R-MS) and Sen. Amy Klobuchar (D-MN) passed in the Senate!
This means that – thanks to the leadership of our Senate champions and our Duchenne…Continue
Added by Annie Kennedy on August 3, 2017 at 12:33pm — No Comments
Added by Abby Bronson on July 31, 2017 at 10:46am — No Comments
Yesterday the Senate voted 51-50 to move forward on healthcare reform legislation -- but no specific bill was actually voted on. The Senate has committed to passing something this week. Once passed, the Senate bill will then have to return to the House for passage or conferencing (if it differs from…Continue
Added by Annie Kennedy on July 26, 2017 at 9:00am — No Comments