Marisa Wexler, MS, senior science writer —

Marisa holds a Master of Science in cellular and molecular pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. Her areas of expertise include cancer biology, immunology, and genetics, and she has worked as a science writing and communications intern for the Genetics Society of America.

Articles by Marisa Wexler

Del-desiran for DM1 wins FDA’s breakthrough therapy designation

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to delpacibart etedesiran (del-desiran, previously called AOC 1001), an investigational treatment for myotonic dystrophy type 1 (DM1) that’s about to enter Phase 3 clinical testing. The FDA gives the designation to experimental therapies that have the…

Diagnostic testing IDs 2 gene mutations causing LGMD type R1

Diagnostic testing of individuals in India suspected of having limb-girdle muscular dystrophy (LGMD) revealed two novel mutations in the calpain-3 gene, known as CAPN3, that were found to cause the common subtype R1, known as LGMDR1. These findings were detailed in a new study reporting the results of testing…

Mitochondrial dysfunction may be a treatment target in DM2

In myotonic dystrophy type 2 (DM2), muscle cells have dysfunctional mitochondria, the cell’s so-called powerhouse that’s key to energy generation. The results imply that treatments to boost mitochondrial function could be beneficial in DM2, the researchers said in “Multi-level profiling unravels mitochondrial dysfunction in myotonic dystrophy…

Exon-skipping AOC 1044 gets FDA rare pediatric disease status

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to AOC 1044, an experimental therapy for people with Duchenne muscular dystrophy (DMD) caused by mutations that are amenable to exon 44 skipping. The FDA gives the designation to treatments with the potential to improve care…