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Duvyzat now available to DMD patients 6 and older in US
Duvyzat (givinostat), a newly approved treatment for Duchenne muscular dystrophy (DMD), is now available in the U.S., according to an announcement from ITF Therapeutics, which will be selling the therapy. “Following the FDA [Food and Drug Administration] approval of Duvyzat, our team has been focused on making this new treatment option available to the DMD…
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PepGen Announces Positive Data from Phase 2 Clinical Trial of PGN-EDO51
PepGen Inc. has shared positive clinical data from the first dose cohort (5 mg/kg) of PGN-EDO51, the company’s lead investigational candidate for patients with Duchenne whose mutations are amenable to exon 51-skipping. PGN-EDO51 is an PMO-exon skipping therapy bound to a peptide which improves uptake into muscle cells, and targets those amenable to exon 51…
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Finding Belonging and Fostering Community
While no one asks to be part of this community, and entering the world of Duchenne and Becker can be overwhelming, you are not alone. During his keynote session at PPMD’s 30th Annual Conference, Brandon Kozar, PhysD, MBA, from Nationwide Children’s Hospital, emphasized the importance of inclusion and feeling a sense of belonging for individuals…
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Pfizer Discontinues Development of Investigational Mini-Dystrophin Gene Therapy
PPMD is disappointed to learn that Pfizer Inc. has officially discontinued development of the company’s investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec. This announcement follows Pfizer’s June 2024 update on the Phase 3 CIFFREO study evaluating the drug in ambulatory patients with Duchenne ages 4 to 7. In June 2024, Pfizer shared that the study did…
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Pfizer Duchenne Program Discontinuation
Pfizer Duchenne Program Discontinuation: Pfizer has officially decided to discontinue their microdystrophin gene therapy program for Duchenne, after the sad announcement last month that the Phase 3 trial failed to meet its primary endpoint. While this is not the outcome we had hoped for, we thank Pfizer for its commitment to sharing the data so…
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Response to the July 29th STAT Article
A brief history: During and since 2016, there has been frustration in this community regarding drug development. Fast forward: PPMD strives for both accuracy and transparency in all information we release. We made the decision to edit and ultimately remove the Research Row: Gene Therapy – Current and Evolving Landscape panel recording in response to…
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New gene therapy approach shows promise for Duchenne muscular dystrophy
Researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new treatments for people with Duchenne muscular dystrophy (DMD).
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New study may help more DMD patients be included in clinical trials
Most children with Duchenne muscular dystrophy (DMD) start experiencing notable issues with arm function and breathing ability before they lose the ability to walk, a new study reports. The findings have major implications for clinical trials testing treatments that aim to boost arm and/or lung function in DMD. Traditionally, such studies have enrolled only patients…
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As a caregiver for my sons with DMD, how do I know what is right?
Wrestling videos were the soundtrack to my morning earlier this week. I was staring at a beige wall void of anything except a small white clock marking the time and the black screen of a television no one uses. Sitting in the infusion center at our local hospital, I thought about how much time my…
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Making it Work: Practical Tips for Enhancing Quality of Life
It’s important to ensure that even within the context of Duchenne/Becker, kids are doing things that kids should be doing, adults are doing things that adults should be doing, and families can lead the lives they want to lead. Together with PPMD, your community, and your care team, it’s all about figuring out how we…
