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Newborn Screening for Duchenne: PPMD’s Collaborative Future Planning Efforts
On Saturday, June 8th, PPMD convened a vital meeting of physicians, physical therapists, genetic counselors, data scientists, and other key opinion leaders to discuss the development of data infrastructure and care planning for newborns that screen for Duchenne through state mandated programs. This meeting featured representatives from Ohio, Minnesota, and New York—the three states that…
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FDA approves first generic of Emflaza oral suspension for DMD
The U.S. Food and Drug Administration (FDA) has approved the first generic version of Emflaza (deflazacort) oral suspension for the treatment of people with Duchenne muscular dystrophy (DMD). Called deflazacort oral suspension, the medication was approved for DMD patients 5 and older, a slightly older population than the original medication, which is approved for patients…
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I am always learning more about life with Duchenne
When three of my sons — Max, 18, Rowen, 15, and Charlie, 13 — were diagnosed with Duchenne muscular dystrophy between July 2010 and August 2011, my learning curve was practically a vertical line. When the diagnosing doctor first said “Duchenne,” it was the first time I had heard it. My initial question was how to…
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Build Your Own Care Binder at PPMD’s 30th Annual Conference
PPMD is excited to share that at our 30th Annual Conference, taking place June 27-29, 2024, in Orlando, Florida, the PPMD Care team will host a “DIY Care Binder” station where families can create a personalized binder filled with important information regarding Duchenne/Becker care to help you navigate routine care and emergencies. A diagnosis of…
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Cranbury Pharmaceuticals (Tris Pharma) Receives FDA Approval for First Generic Version of Emflaza® (deflazacort) for Duchenne
Cranbury Pharmaceuticals, a subsidiary of Tris Pharma, today announced the U.S. Food and Drug Administration (FDA) approved the Abbreviated New Drug Application (ANDA) for the first generic version of Emflaza® oral suspension (deflazacort) for the treatment of Duchenne. The generic deflazacort oral suspension, a corticosteroid indicated to treat Duchenne in patients five years of age…
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Pfizer Shares Update on Phase 3 Study of Investigational Mini-Dystrophin Gene Therapy
PPMD is deeply disappointed to share that Pfizer Inc. announced today that CIFFREO, a Phase 3 randomized, double-blind, placebo-controlled study evaluating the company’s investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec, in ambulatory patients with Duchenne did not meet its primary endpoint of improvement in motor function among boys 4 to 7 years of age treated with…
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While we’re all on unique paths, I welcome input from my community
Over the past couple months, I’ve shared my decision to try physical therapy in an attempt to slow the progression of my facioscapulohumeral muscular dystrophy (FSHD). I also shared that I haven’t seen a lot of benefit from the physical therapy, at least in terms of regaining much strength. At this point, I’m continuing to…
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Pitolisant found to reduce fatigue, daytime sleepiness in DM1 patients
After nearly three months of treatment with pitolisant, adults with myotonic dystrophy type 1 (DM1) experienced a reduction of excessive daytime sleepiness (EDS) and fatigue. That’s according to data from the Phase 2 trial (NCT04886518), which is testing the safety and efficacy of Harmony Biosciences’ therapy in DM1 patients, ages 18 to 65, with moderate…
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Pfizer Provides Update on Phase 3 Study of Investigational Gene Therapy for Ambulatory Boys with Duchenne Muscular Dystrophy
As a community, we are crushed to hear the news from Pfizer that their Phase 3 mini-dystrophin gene therapy trial in Duchenne failed to meet its primary endpoint. Individuals, aged 4 to 7 years old, treated with the experimental gene therapy did not show significant improvement in motor function, as measured by the North Star…
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Consensus Statement Featuring Expert Insights from PPMD’s Certified Duchenne Care Centers on Access to Novel Therapies for Duchenne Published
PPMD is excited to share that a consensus statement providing insights from clinicians who care for patients with Duchenne into the current therapeutic landscape and access to novel therapies for Duchenne has been published in the Annals of Child Neurology Society. The landscape of approved therapies in Duchenne is rapidly changing with eight approved therapies,…
