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PPMD Urges FDA to Conduct a Full Review of Ataluren (Translarna) Following Resubmission of NDA
PTC Therapeutics has shared that it has resubmitted its New Drug Application (NDA) to the FDA for review and potential approval of ataluren (Translarna). Ataluren is an oral small molecule treatment that allows for stop-codon read through to produce dystrophin in patients with nonsense mutations or ‘periods in the middle of their genetic sentence.’ Up…
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Avidity Biosciences Announces Positive AOC 1044 Data
Avidity Biosciences, which received early funding from CureDuchenne, announced positive initial data from their EXPLORE44 Trial for individuals with Duchenne amenable to skipping exon 44. Their experimental exon-skipping agent, AOC 1044 (del-zota), at 5 mg/kg increased exon 44 skipping by an average of 37% (from 7% at baseline to 44%) and increased average dystrophin levels…
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The dance floor at my daughter’s wedding had room for all
I’m officially a mother-in-law! That’s a new title for me. I’m a mom to the seven children I share with my husband, Jason. I’m also a special needs mom and warrior mama to my three sons living with Duchenne muscular dystrophy (DMD). And now I’m also a mother-in-law to my daughter Lexi’s husband, Nick. They…
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Avidity Biosciences Announces Positive Data From Phase 1/2 EXPLORE44™ Trial of AOC 1044 in Individuals Amenable to Exon 44 Skipping
Avidity Biosciences, Inc. today announced positive data from the company’s Phase 1/2 EXPLORE44 clinical trial of AOC 1044, an exon skipping therapy targeting those amenable to exon 44 skipping. Avidity also announced the approved international nonproprietary name of AOC 1044 as delpacibart zotadirsen, abbreviated as del-zota.. The randomized, double-blind, placebo-controlled Phase 1/2 EXPLORE44 trial of…
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WATCH: Edgewise Therapeutics – Clinical Trials in Becker Muscular Dystrophy (Webinar)
Edgewise Therapeutics recently joined PPMD for a webinar to provide the Becker community information about the company’s clinical trials for Becker. Edgewise’s Chief Scientific Officer and Chief Medical Officer, shared two year results from the completed Phase 1 ARCH study in adults with Becker and more information about their current enrolling trial, GRAND CANYON, a…
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I’m at a crossroads in life, once again
Starting last week, I’ve been trying to get my life back on track after enduring a few difficult months. As I recently shared, life has been challenging since I left my job in April. Then, in May, I faced an unexpected hospitalization, and June brought relationship tension. In July, my mental health issues resurfaced, exacerbating…
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Phase 1/2 update ‘encouraging’ for DMD gene therapy RGX-202
The experimental gene therapy RGX-202 has been found to be well tolerated at a high dose, with biomarker data indicating it is working as designed to increase production of the microdystrophin protein in boys with Duchenne muscular dystrophy (DMD). That’s according to updated interim findings from the ongoing Phase 1/2 AFFINITY DUCHENNE clinical trial (NCT05693142),…
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Your Citizen Science At Work
By sharing your anonymous data through The Duchenne Registry, you are strengthening the power of a 15-year-old network of patient-powered data that is used to improve care for people living with Duchenne and increase our understanding of the disorder. Data is shared with researchers, fueling the fight to end Duchenne and helping to speed the…
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PGN-ED051, skipping exon 51, showing benefits in Phase 2 trial
A low dose of PGN-ED051, PepGen’s investigational exon 51-skipping therapy, safely increased dystrophin protein levels in people with Duchenne muscular dystrophy (DMD), according to early Phase 2 clinical trial data. The therapy’s effects either were comparable to or greater than what has been observed in studies of other exon 51-skipping therapies at a similar dose…
