August 2017 Blog Posts (8)

FDA Announces Approval of First Gene Therapy in the United States

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, remarked on the FDA's approval of the first gene…


Added by Abby Bronson on August 31, 2017 at 1:00pm — No Comments

Care Complications – Going Beyond the More Common Elements of Care in Duchenne

Every year at PPMD's Annual Connect Conference, we try to put together the best topics, with the most informed speakers, to address elements of care at…


Added by Kathi Kinnett on August 28, 2017 at 11:30am — No Comments

Webinar Q&A: Gene Therapy for Duchenne

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.

Earlier this month, PPMD hosted a webinar with Lee Sweeney, PhD (University…


Added by Abby Bronson on August 23, 2017 at 8:00pm — No Comments

How You Can Participate in the Ataluren Ad Comm

The Duchenne community is now preparing for the September 28 Advisory Committee Meeting for PTC’s therapy ataluren. Whether submitting written testimony and/or attending the Ad Comm in person, there are multiple…


Added by PPMD on August 22, 2017 at 11:00am — No Comments

PPMD's Bone Health Workshop Published

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 confusing and often neglected aspect of …


Added by Kathi Kinnett on August 14, 2017 at 10:00pm — No Comments

Understanding Clinical Trials in Duchenne

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 another path to achieve our goal and it allows us to look for…


Added by Ann Lucas on August 7, 2017 at 2:00pm — No Comments

PDUFA VI Passes the Senate – Now Moves to President’s Desk to be Signed Into Law

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…


Added by Annie Kennedy on August 3, 2017 at 8:30pm — No Comments

The BENEFIT Act Passes the Senate!

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 community – the agency’s signature tool for evaluating…


Added by Annie Kennedy on August 3, 2017 at 12:33pm — No Comments

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