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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - February 15, 2017 Category: Neurology Source Type: news
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! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - February 14, 2017 Category: Neurology Source Type: news
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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - February 13, 2017 Category: Neurology Source Type: news
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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - February 9, 2017 Category: Neurology Source Type: news
PPMD to Host Forum on Access to Emerging Therapies on February 13th, 2017
Following decades of strategic advocacy, research investment, and biopharmaceutical development, the U.S. Duchenne community recently arrived in the post-approval space and is poised to receive additional novel therapies from a robust innovation pipeline. PPMD is engaged on behalf of families nationwide as industry sponsors work with the FDA to navigate the critical regulatory steps. We are also working now within the access environment to help ensure that families have timely access to these disease-modifying medications. It is our hope that this Forum will be an important step in helping to continue to empower our Duchen...
Source: Parent Project Muscular Dystrophy - February 8, 2017 Category: Neurology Source Type: news
[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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - February 6, 2017 Category: Neurology Source Type: news
[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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - February 3, 2017 Category: Neurology Source Type: news
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! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - February 2, 2017 Category: Neurology Source Type: news
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 (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - February 1, 2017 Category: Neurology Source Type: news
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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - February 1, 2017 Category: Neurology Source Type: news
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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - February 1, 2017 Category: Neurology Source Type: news
[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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - January 27, 2017 Category: Neurology Source Type: news
[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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - January 20, 2017 Category: Neurology Source Type: news
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! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - January 19, 2017 Category: Neurology Source Type: news
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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - January 18, 2017 Category: Neurology Source Type: news
