March 2018 Blog Posts (10)

End Duchenne Tour Recap: Grand Rapids, MI

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.…


Added by Ryan Fischer on March 28, 2018 at 4:30pm — No Comments

Center Directors Publish Collective Statement Regarding Patient Access to Approved Therapies

PPMD is proud to announce the publication of a collective statement regarding patient access to approved therapies from the center directors of our Certified Duchenne Care Centers.

There are many ongoing treatment trials for Duchenne and of…


Added by Kathi Kinnett on March 27, 2018 at 9:00am — No Comments

[WEBINAR] What is a Master Protocol?

PPMD fights to end Duchenne for every single family. As part of our mission, we believe that we must continually work to identify and explore innovative opportunities that may accelerate the drug development and clinical trial process, getting effective therapies to individuals with Duchenne as quickly as possible.

Many of the families in our…


Added by PPMD on March 26, 2018 at 12:00pm — No Comments

Santhera Shares Duchenne Community Update on SIDEROS Trial

Santhera has provided a community update, including the latest news on their SIDEROS trial, currently recruiting. Click here to view the update.…


Added by PPMD on March 23, 2018 at 10:02am — No Comments

Sarepta Therapeutics Announces Launch of Route 79, The Duchenne Scholarship Program

Sarepta Therapeutics announced the launch of Route 79, The Duchenne Scholarship Program, an annual scholarship for students diagnosed with Duchenne. The Route 79 program is designed to help students with Duchenne pursue their post-secondary educational goals. Scholarships of up to $10,000 will be awarded to 10 individuals chosen by an independent committee of Duchenne community members based on an applicant’s…


Added by PPMD on March 20, 2018 at 10:54am — No Comments

Solid Biosciences Announces Clinical Hold On SGT-001 Phase I/II Clinical Trial For Duchenne

Solid Biosciences announced that the FDA has placed their Phase I/II clinical trial for SGT-001, IGNITE DMD, on Clinical Hold following a serious adverse event that occurred in the first patient dosed, a non-ambulatory adolescent. The patient was admitted to the hospital, received treatment, and is home with his…


Added by PPMD on March 15, 2018 at 8:30am — No Comments

ADD YOUR VOICE: Participate in PPMD’s Gene Therapy Survey

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

Roche Shares Update on RG62026 Phase 2/3 Study

Roche has provided an update to the community regarding the paused recruitment of RG6206, an investigational anti-myostatin adnectin, in ambulatory boys with Duchenne. The company hopes to restart recruitment in the second quarter of this year.

Read the update from Roche: …


Added by PPMD on March 14, 2018 at 11:00am — No Comments

Duchenne Pulmonary Awareness Video Series

Pulmonary management in Duchenne (all of the care involved with breathing and coughing) can be confusing and difficult to understand. There are many terms, abbreviations, and procedures that all sound very complicated. PPMD – with support from Santhera Pharmaceuticals and input from a panel of parents,…


Added by Kathi Kinnett on March 13, 2018 at 9:30am — No Comments

Add Your Voice to the Historic Compass Meeting

There are hardly words to capture the impact our unified community had on Monday. We had traveled from all over the nation to convene with federal policymakers, industry partners, and payers for the Duchenne Patient-Focused Compass Meeting: Navigating our Pathway Forward. Nested within PPMD’s…


Added by Pat Furlong on March 8, 2018 at 2:00pm — No Comments

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