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