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

Experimental Gene Therapy AT845 Shows Promise in Animal Study

An investigational gene therapy called AT845 could effectively increase the activity of an enzyme that’s deficient in mouse and nonhuman primate models of Pompe disease, according to a study. The research also highlights potential complications that can occur when gene therapies designed for use in humans are tested in…

Applications for AT-GAA Under Review in Europe

The European Medicines Agency has accepted applications from Amicus Therapeutics seeking approval of AT-GAA, a two-component investigational therapy for the treatment of Pompe disease. “The acceptance of these filings is an important step forward for people living with Pompe disease and their families in Europe. Patients need new…

Nexviazyme Approved in Canada as Next-gen ERT for Late-onset Pompe

Nexviazyme (avalglucosidase alfa), a next-generation enzyme replacement therapy, has been approved by Health Canada for people with late-onset Pompe disease ages 6 months and older. “The Health Canada approval of Nexviazyme is an important milestone for Canadian Pompe patients and may represent a new standard of care,” Mark…

CPAP Training May Help Children Improve Speech, Small Study Shows

Training with continuous positive airway pressure therapy, known as CPAP, may help to normalize speech abnormalities in children with Pompe disease, a small pilot study shows. Certain outcomes, including the speech intelligibility score (SIS), “were significantly improved” over the course of the six-month study, but as two of the…

FDA to Decide on AT-GAA for Late-onset Pompe Around Mid-2022

The U.S. Food and Drug Administration (FDA) is reviewing applications from Amicus Therapeutics asking for the approval of AT-GAA as a treatment for late-onset Pompe disease (LOPD). Amicus initiated a rolling application for AT-GAA late last year, which allowed the company to submit portions of its application as…

Early ERT Use May Lessen Hearing Loss in Infantile-onset Pompe

Starting treatment with enzyme-replacement therapy (ERT) in the first days of life appears to lessen the risk of hearing impairments in children with infantile-onset Pompe disease (IOPD), researchers report. “Our study may enhance awareness of early intervention before hearing-related morbidities can develop in patients with IOPD,” its researchers…