AOC 1020 Now on FDA’s Fast Track as FSHD Treatment

The U.S. Food and Drug Administration (FDA) has given fast-track designation to AOC 1020, an investigational therapy for facioscapulohumeral muscular dystrophy (FSHD) developed by Avidity Biosciences. This designation allows more frequent interactions between a company and the regulatory agency, with the goal of speeding the development and review…

On Dec. 4, I performed British singer-songwriter Calum Scott’s 2017 pop ballad “You Are the Reason” at a live caroling session with ART:DIS, the first organization in Singapore to pioneer the artistic development of members of the disabled community. The performance was part of the annual Enabling Lives…

Pelvic floor disorders, including urinary and anal incontinence, are common in women with myotonic dystrophy type 1 (DM1) and a cause of significant distress, according to a recent study in Canada. “This study highlights the importance of screening these symptoms in a clinical setting and will help develop interventions…

Before three of my sons were diagnosed with Duchenne muscular dystrophy (DMD), I didn’t know what I didn’t know. After that, my learning curve went straight up. I still needed to learn the amount of advocacy work it takes to be a parent to a child (or…

Entrada Therapeutics has selected ENTR-601-45 as an investigational therapy candidate for people with Duchenne muscular dystrophy (DMD) who have mutations amenable to exon 45 skipping. The company is planning to file an Investigational New Drug application in the second half of 2024. These applications are used to request…

The  U.S. Food and Drug Administration (FDA) is reviewing vamorolone for Duchenne muscular dystrophy (DMD) and has set a target action date of Oct. 26 for its decision on whether to approve the dissociative corticosteroid treatment. The regulatory agency accepted a new drug application (NDA) for the experimental…

I do my best to try in life, but I’m not perfect by any means. That’s me. I hope that is how I come across in this column. However, I never want to make life in a Duchenne muscular dystrophy (DMD) family sound easy, perfect, or Instagram-worthy. Truthfully, if…

Satellos Bioscience has designated SAT-3153 as a potential muscle regeneration treatment for Duchenne muscular dystrophy (DMD). The therapeutic candidate now will be tested in pre-investigational new drug (pre-IND) enabling studies intended to provide evidence to obtain regulatory approval for conducting a clinical trial. “We are thrilled to be in…

The approved antiparasitic medicine pentamidine, loaded into biocompatible and biodegradable nanoparticles, successfully eased signs of myotonic dystrophy type 1 (DM1) in cell-based models, a study shows. The researchers noted that these findings show the potential of nanoparticles to deliver therapeutic agents to treat muscle cells in people with DM1…

Is it necessary to write about my family and three children with Duchenne muscular dystrophy (DMD)? I have always felt that it is. However, sometimes, especially on social media, I am questioned and criticized when I share certain challenging aspects of life. “Why does she have to share that?”…