We are pleased to report our final numbers for the FY19 letters.
In total, 19 Senators signed onto the Senate FY19 letter, joining 51 members of the House. That makes a solid 70 members of Congress (House and Senate combined) sending a strong message of support behind Duchenne research, public health, drug development, and patient support initiatives. They provide strategic direction for the federal agencies that touch Duchenne. Given the…Continue
Added by Ryan Fischer on April 16, 2018 at 11:30am — No Comments
PPMD was excited to head to the Midwest for the second stop on the 2018 End Duchenne Tour. The event took place at Western Michigan University in Grand Rapids, Michigan, in partnership with Team Joseph, Little Hercules Foundations, and Noah’s Feat.…Continue
Added by Ryan Fischer on March 28, 2018 at 4:30pm — No Comments
Patient-Focused Drug Development in action
Companies developing gene therapies, the FDA, and other stakeholders are interested in the treatment priorities of the Duchenne community. Because of this, PPMD has collaborated with RTI International, with financial support from Solid Biosciences and…
Added by Ryan Fischer on March 14, 2018 at 1:30pm — No Comments
PPMD kicked off the 2018 End Duchenne Tour with a stop in our very own backyard - Goryeb Children's Hospital in Morristown, New Jersey! PPMD was extremely grateful to partner with Michael's Cause, Pietro's Fight, Powers Promise, and Ryan's Quest on…Continue
Added by Ryan Fischer on February 15, 2018 at 2:30pm — No Comments
The PPMD Advocacy Conference registration is live! Join us March 4-6 in Washington DC to advocate for Duchenne directly with Members of Congress.
Added by Ryan Fischer on January 23, 2018 at 4:50pm — No Comments
Navigating access to approved therapies can be both time consuming and daunting. The time between approval by FDA to the day of a patient’s first commercial dose can vary depending on which insurance you have, your age and disease progression, and even where you live. With two…Continue
Added by Ryan Fischer on November 20, 2017 at 8:00pm — No Comments
Boston made for a great location for our October Every Single One Tour stop. Nothing beats the northeast in the fall. Boston is home to a number of great Duchenne organizations, a network of active families, and where most of the drug companies…Continue
Added by Ryan Fischer on October 30, 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
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
The newest version of the GOP HealthCare Bill is coming to a vote by 1:30 PM EDT this afternoon!Continue
Added by Ryan Fischer on May 4, 2017 at 12:00pm — No Comments
Added by Ryan Fischer on December 7, 2016 at 11:04am — No Comments
We need you to call your two US Senators TODAY with one simple message – VOTE YES ON 21st CENTURY CURES!!
With just days remaining in the current Congress, this bill represents the extraordinary efforts of our PPMD community’s work and outreach with Congress. We are delighted to report that the version in front…Continue
Added by Ryan Fischer on December 5, 2016 at 10:07am — No Comments
You are invited to join us February 12-14, 2017 in Washington D.C. for what is going to be another critical year in Duchenne advocacy.
2016 marked a tremendous…Continue
Added by Ryan Fischer on October 20, 2016 at 1:00pm — No Comments
Added by Ryan Fischer on August 23, 2016 at 12:00pm — No Comments
Thanks to your advocacy, this week we learned that all of the Duchenne priorities we advocated for this year with Members of Congress are one step closer to being included in next year’s budget.
Whether you traveled to DC for face-to-face meetings with members, or advocated from home through emails, phone calls, and social media – your actions…Continue
Added by Ryan Fischer on June 10, 2016 at 12:00pm — No Comments
Bipartisan Group of Senators Urge FDA to Utilize All Available Tools to Speed Access to Promising Duchenne Therapies
On Friday April 15th, U.S. Senators Roger Wicker, (R-MS) and Amy Klobuchar, (D-MN), sent a…Continue
Added by Ryan Fischer on April 18, 2016 at 9:44am — No Comments
Earlier this year, 109 members of the House of Representatives sent a powerful letter to the FDA urging them to “utilize all available tools, resources, and authorities” in reviewing a treatment for Duchenne muscular dystrophy. Congressional champions Congressmen Mike Fitzpatrick (R-PA), Bill Keating (D-MA), and Peter King (R-NY) led this incredible bipartisan letter.…Continue
Added by Ryan Fischer on April 5, 2016 at 11:30am — No Comments
We are just a few short weeks from the FDA Advisory Committee Meeting for eteplirsen on April 25th. PPMD is part of a united coalition of Duchenne organizations (MakeDuchenneHistory Coalition) working together to ensure we make the largest possible impact on the advisory committee and the…Continue
Urge your Lawmakers to Sign the FY 17 Duchenne Appropriations Letter!
Today and tomorrow, scores of PPMD advocates will be on Capitol Hill to urge their Senators and Members of Congress to continue supporting vital efforts that will move us closer to ending…Continue
Added by Ryan Fischer on February 22, 2016 at 1:43pm — No Comments
Thanks to this incredible community urging lawmakers, well over 100 Members of Congress signed and submitted a letter …