PPMD Designates Lurie Children's a Certified Duchenne Care Center
PPMD is excited to announce our 14th Certified Duchenne Care Center -- Ann & Robert H. Lurie Children’s Hospital of Chicago!
Upcoming Webinar 2/22: NS Pharma's Exon 53 Skipping Program
Join PPMD and NS Pharma as we host a webinar Wednesday, February 22 at 1:00 PM EST to to provide an update on trial recruitment for NS Pharma's Exon 53 Skipping Program.
PPMD Recommends Marathon Convene Broad Array of Duchenne Stakeholders to Discuss Re-evaluation of Deflazacort Pricing
In light of the pause in commercialization of EMFLAZA™ (deflazacort) by Marathon Pharmaceuticals earlier this week, PPMD recommends that Marathon seek input from a broad array of stakeholders including Duchenne clinicians, health insurance plans, pharmacy benefit managers, and Duchenne advocacy groups.
End Duchenne eNews: Raising Our Voices for Every Single One
Catch up on the latest research and community updates in this month's End Duchenne eNews.
Urge Congress to Protect Duchenne Community's Healthcare Priorities
This week, PPMD advocates from across the country are storming Capitol Hill to ensure legislators make Duchenne and rare disease a priority.
This year’s agenda includes urging Congress NOT to repeal the Patient Protection and Affordable Care Act until there is a guarantee that people with Duchenne will continue to have access to equal or better coverage. Please take a moment today to tell Congress why this matters so much to our community!
Marathon Pharmaceuticals Update on EMFLAZA™ (deflazacort)
Marathon has announced that they will pause the commercialization of their recently FDA-approved steroid EMFLAZA™ (deflazacort).
This pause will allow Marathon to consider the feedback they have received from the community since last week’s announcement regarding pricing and potential for combining this therapy with other approved drugs in the future. Marathon has assured families that during this pause, access to deflazacort will not be disrupted.
Deflazacort Approved in the U.S. for Duchenne
Today, the FDA approved EMFLAZA™ (deflazacort) for Duchenne. This is the first approval of a steroid for Duchenne and a therapy that applies to people with Duchenne 5 years of age and older, regardless of genetic mutation. Representatives from Marathon Pharmaceuticals will be joining us for a webinar Friday, February 10th at 12 PM EST to provide information about EMFLAZA’s FDA-approved label, patient support services, and access to this medication.
PPMD to Host Forum on Access to Emerging Therapies
PPMD is hosting the Access to Emerging Therapies Forum on February 13th, 2017 for our patient community and relevant stakeholders. Our presenters include policy experts who will present on the various points on the access continuum.
[Webinar Recording] MoveDMD Trial: Catabasis Provides Update
The Catabasis team recently joined us to discuss what they have learned from Part B of the MoveDMD® trial of edasalonexent (CAT-1004) in Duchenne and what comes next in the MoveDMD program.
[Webinar Recording] Mission DMD: FibroGen’s Anti-Fibrosis Program
FibroGen recently joined us for a webinar discussion on the company's investigational drug Pamrevlumab (FG-3019), along with FibroGen’s Mission DMD program that is currently being conducted to investigate Pamrevlumab in Duchenne.
Registration for our 2018 Walt Disney World Marathon Weekend team is now open!
2018 marks the 25th anniversary of the Full Marathon, with lots of other fun race distances available too. It's the biggest and best marathon weekend of the year for our Run For Our Sons team, and spots are filling up fast. Don't miss out on this magical weekend!
PPMD’s Duchenne Drug Development Roundtable: Bringing Industry Together to Accelerate Research
Today is the first of PPMD's Duchenne Drug Development Roundtable (DDDR) three-meeting series to address clinical trial challenges and opportunities within our Duchenne space.
PPMD Awards $250,000 Grant to UT Southwestern Medical Center to Explore CRISPR/Cas9 Technology
This grant, part of PPMD’s Gene Transfer Initiative, will support Dr. Olson’s ongoing study of CRISPR/Cas9 technology as a potential treatment for Duchenne
[Webinar Recording] Summit's Utrophin Modulation Program
Summit joined us earlier this week for an educational webinar on utrophin modulation, an approach for Duchenne that has the potential to treat all patients.
[Webinar Recording] Vamorolone (VBP15) Clinical Trials in Duchenne
ReveraGen BioPharma CEO Dr. Eric Hoffman joined us earlier this week for a webinar discussion on vamorolone (VBP15) clinical trials in Duchenne.
PPMD Designates Yale New Haven Children's Hospital a Certified Duchenne Care Center
PPMD is excited to announce our 13th Certified Duchenne Care Center (CDCC) the Yale New Haven Children's Hospital's Muscular Dystrophy Program!
PPMD Awards $600,000 Grant to NJIT & Talem for Next Phase of Upper Extremity Exoskeleton
PPMD has awarded a $600,000 grant to the New Jersey Institute of Technology (NJIT) and Talem Technologies as part of our ongoing exploration of robotic technology to assist people living with Duchenne.
New Resource to Help Navigate EXONDYS 51 Access
Dr. Craig McDonald of UC Davis has shared data and an accompanying presentation to serve as a resource for families who have received an EXONDYS 51 denial from their insurance company and are working to appeal the decision. You should insist that both your clinician and insurance company review this data and presentation.
New Research & Clinical Trial Tools on DuchenneConnect
We are excited to announce three new tools now available on PPMD's DuchenneConnect! These tools are meant to help you in your journey as you gather information around potential research studies and potential new exon skipping therapies.
PPMD Awards $2.2 Million Grant to Explore Gene Therapy in Duchenne
PPMD has announced a $2.2 million dollar grant to Dr. Jerry Mendell, Dr. Louise Rodino-Klapac (co-PI), and Nationwide Children’s Hospital. This grant is the first project of a new Gene Transfer Initiative PPMD is launching this month that will include the work Dr. Mendell and Dr. Rodino-Klapac are doing, as well as a deeper exploration of CRISPR/Cas9 technology as a potential therapeutic approach to treating Duchenne.