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Adjusting my definition of what it means to be a man
Kevin Schaefer, the associate director of community content at Bionews, the parent company of this website, recently shared a thought-provoking article from Psychology Today titled “How Chronic Illness and Masculinity Intersect.” It has me thinking about what kind of man I am and want to be. The article notes that “changes caused by chronic illness…
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SAT-3247 for DMD wins FDA rare pediatric disease designation
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to SAT-3247, an oral treatment candidate for Duchenne muscular dystrophy (DMD) that ultimately aims to slow the progression of the genetic condition. The FDA awards this status to investigational medicines that are designed to treat serious, rare diseases that mainly affect children.…
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Finding Support to Manage Behaviors in Duchenne and Becker
Research has shown that dystrophin is usually present in the brain as well as in the muscles. Scientists believe that when dystrophin is missing or partially functional, like in Duchenne and Becker, brain neurons may not function as well as they should. This can lead to learning and behavior challenges for some children. While not…
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Solid Biosciences has begun dosing patients in their clinical study of their next-generation gene therapy, SGT-003 for those living with Duchenne.
Letter to the Duchenne Community – INSPIRE DUCHENNE update Dear Duchenne Community, We are writing to share an important update on INSPIRE DUCHENNE, our Phase 1/2 clinical trial of SGT-003 for the treatment of patients living with Duchenne. At Solid Biosciences, our mission is driven by the needs and hopes of the Duchenne community, and we…
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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 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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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 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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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…
