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
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…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 confusing and often neglected aspect of …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 another path to achieve our goal and it allows us to look for…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 community – the agency’s signature tool for evaluating…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 the House version). Then it moves to the President's desk to…Continue
Added by Annie Kennedy on July 26, 2017 at 9:00am — No Comments
Today, the Senate is expected to start floor debate and motion for a vote on either the Better Care Reconciliation Act (BCRA), which would repeal and immediately replace the Affordable Care Act (ACA), or the Obamacare Repeal Reconciliation Act (ORRA), which would repeal portions of the ACA after a two-year delay. The Senate is working to pass whichever bill is more likely to attain 50 votes.
The Congressional Budget Office released reports last week that…Continue
Added by Annie Kennedy on July 25, 2017 at 9:05am — No Comments
Transitions are hard. That’s the truth whether you’re talking about a new school year, a new job, or even a new Congress. And, in particular, new Presidential Administrations are always complex political climates in Washington.
But this year, our Duchenne community came together once again and embraced the challenge of this latest transition. With so many new Members of Congress and Federal Agency leadership in flux, we knew that our 2017 February…Continue
Added by Annie Kennedy on July 19, 2017 at 12:00pm — No Comments
In April 2016, PPMD convened a workshop to assess the measures used to evaluate pulmonary strength, air flow and lung volumes. Thanks to the tireless effort of Dr. Jonathan Finder, this summary has now been published.…Continue
Added by Kathi Kinnett on July 17, 2017 at 9:35am — No Comments
Sadly, I never had the honor of meeting PJ Nicholoff, but I’ve met his parents. And if PJ was anything like his father, Brian, he was vibrant, compassionate, caring, engaged and tenacious!
Added by Kathi Kinnett on July 16, 2017 at 8:30pm — No Comments
Today Parent Project Muscular Dystrophy (PPMD) named Gillette Children's Specialty Healthcare Neuromuscular Clinic (Gillette) our 16th Certified Duchenne…Continue
Added by PPMD on July 13, 2017 at 11:21am — No Comments
Added by PPMD on June 30, 2017 at 1:49pm — No Comments
Today, PPMD is convening medical experts from around the world for a workshop exploring inflammation and immunity in Duchenne on the eve of our 23rd Annual Connect Conference.
In January, PPMD organized a Critical Path Innovation Meeting with the FDA focused on…Continue
Added by PPMD on June 28, 2017 at 2:19pm — No Comments
An Overview of Your Impact in Action
We are less than a week away from PPMD’s Annual Connect Conference, where researchers and industry from around the world will provide…Continue
Added by Abby Bronson on June 23, 2017 at 2:00pm — No Comments
Navigating life with Duchenne can be daunting and time consuming for families. We rely on each other to share wisdom and information in order to make the process smoother and life a bit easier. The best guidance we can get is from those who have been through a given situation, sharing what they’ve learned to help others navigate resources that exist.
PPMD strives to find…Continue
Added by Ryan Fischer on June 21, 2017 at 10:00am — No Comments
HOLA MI HIJO JOSE DAVID NECESITA DE SU APOYO SOMOS UNA FAMILIA DE ESCASOS RECURSOS ECONOMICOS DE CARTAGENA COLOMBIA ,ERAN DOS HERMANOS CON DUCHENNE YA FALLECIO UNO EL 1 DE DICIEMBRE DEL 2011 A LOS 17 AÑOS .POR LA FALTA DE UNA BUENA ALIMENTACION Y LA CASA EN MAL ESTADO LE DIO UNA FUERTE NEUMONIA EL CUAL LE PROBOCO EL PARO RESPIRATORIO...POR FAVOR AYUDEN A MI OTRO HIJO CON DUCHENNE JOSE DAVID VIAÑE DIAZ A QUE TENGA UNA MEJOR CALIDAD…Continue
Added by ramon ciriaco viaña ortiz on June 16, 2017 at 1:04pm — No Comments
Recently, FDA Commissioner Scott Gottlieb announced that the hiring freeze, instituted on January 23, has been lifted.
This reversal means that the FDA can begin to fill vacancies – needed resources for continued research into rare disease treatments and for the review and approval of new medicines.
PPMD is proud to have been one…Continue
Added by PPMD on June 15, 2017 at 11:53am — No Comments
With Congress not wrapping up spending bills for Fiscal Year 2017 until we were more than 7 months into the fiscal year, the FY 2018 budget and appropriations (funding) cycle will be interesting to say the least. The Trump Administration released a preliminary — or “skinny” budget -- in March but did not release a more complete budget until the end of…Continue
Added by Ryan Fischer on June 15, 2017 at 11:30am — No Comments