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Finding beauty in how Duchenne has shaped our lives
Duchenne muscular dystrophy (DMD) touches every part of my family’s life. Jason, my husband of 23 years, and I have seven children. Three of them — Max, 18, Rowen, 15, and Charlie, 13 — live with DMD. We’re now in the middle of what could be one of the most exciting periods of our family’s…
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REGENXBIO ANNOUNCES NEW POSITIVE DATA FROM AFFINITY DUCHENNE® TRIAL OF RGX-202
Link to press release We are thrilled to share the latest data from RegenXBio’s microdystrophin gene therapy, AFFINITY DUCHENNE®. RGX-202 is a one-time gene therapy for Duchenne designed to deliver a novel microdystrophin gene via AAV8. RGX-202 is differentiated from other microdystrophin gene therapies in that it contains a larger portion of the C-Terminal domain,…
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Pfizer stops DMD gene therapy development after trial failure
Pfizer has discontinued development of fordadistrogene movaparvovec, its investigational gene therapy for Duchenne muscular dystrophy (DMD), after recent Phase 3 trial data indicated a failure to improve motor function in boys with the neuromuscular disease. Patients who have already received the gene therapy in the CIFFREO Phase 3 trial (NCT04281485) or other clinical studies will…
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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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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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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).
