Blog

FDA’s Patient-Focused Drug Development Initiative: What Have We Learned?

PPMD’s SVP of Legislation and Public Policy, Annie Kennedy, joined a distinguished panel discussion convened by FasterCures about the impact of Patient Focused Drug Development on the Duchenne community and how it has become a…

Learn More

Positive Preliminary Results from the First Three Children Dosed in Phase 1/2a Gene Therapy Micro-dystrophin Trial

Sarepta Therapeutics announced that at the Company’s R&D Day, Jerry Mendell, M.D. of Nationwide Children’s Hospital presented positive preliminary results from its Phase 1/2a gene therapy clinical trial assessing AAVrh74.MHCK7.micro-Dystrophin in individuals with Duchenne. Ryan…

Learn More

Join the 2018 #PPMDConference Live Stream!

Next week we will descend on Scottsdale, Arizona for PPMD’s 24thAnnual Conference! With our most expansive agenda to date, including presentations from dozens of researchers, clinicians, and industry partners, we wanted to make this year’s…

Learn More

Solid Biosciences Announces FDA Removes Clinical Hold on SGT-001

PPMD is excited to learn that Solid Biosciences Inc. announced that the FDA has lifted the clinical hold on IGNITE DMD, the Company’s Phase I/II clinical trial for its investigational microdystrophin gene transfer, SGT-001. In…

Learn More

Jumping Jack Events to End Duchenne Turns 15

For 15 years, the Knight family of Westminster, Colorado has been supporting PPMD’s mission by hosting their annual Jumping Jack Events every June for 15 years now! Named after their son, 17-year—old Jack Knight, Jumping Jack Events started as…

Learn More

3rd Annual Kindness Over Muscular Dystrophy Raises $140,000

On June 9, the Curran Family had over 500 attendees and raised over $140,000 at their 3rd Annual Kindness Over Muscular Dystrophy event to benefit PPMD in honor of their son, Conner. The event took…

Learn More

A Game Night to End Duchenne

Game Night to End Duchenne in honor of Henry was a huge success last weekend! Raising close to $50,000 with no overhead costs, it was a night filled with close friends of the family and…

Learn More

End Duchenne Tour Recap: Birmingham, AL

PPMD was very happy to have the opportunity to meet the team from Children’s Hospital of Alabama and all of the families that came out to our End Duchenne Tour stop on June 6 in the…

Learn More

Sarepta Shares Quarterly Update, Including US Clinical Trial Updates

Sarepta has provided another quarterly update to the community, including information on their PPMO (Peptide Conjugated Phosphorodiamidate Morpholino Oligomer), an exon skipping technology designed to efficiently get inside cells, with the goal of producing more dystrophin in…

Learn More

Join the Race to End Duchenne

We are thrilled to announce the launch of the new name and look for our endurance program, Race to End Duchenne. This new name reflects the dynamic nature this program has taken on over the…

Learn More

Join Our Mailing List