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

Next-gen gene therapy SGT-003 named FDA orphan drug

SGT-003, a next-generation gene therapy candidate for Duchenne muscular dystrophy (DMD) being developed by Solid Biosciences, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). The FDA gives this designation to therapies that are designed to improve medical care for rare disorders, specifically defined…

Agamree approved in Europe to treat DMD boys ages 4 and older

The European Commission has approved Agamree (vamorolone), a dissociative corticosteroid — a treatment expected to have fewer side effects than standard steroid medications — for patients ages 4 and older with Duchenne muscular dystrophy (DMD). The decision applies to all member states of the European Union, plus Iceland,…

EDG-5506 wins FDA orphan drug designation for DMD and BMD

The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease designations to Edgewise Therapeutics‘ EDG-5506 as a potential treatment for Duchenne muscular dystrophy (DMD). EDG-5506 also was awarded FDA orphan drug status for Becker muscular dystrophy (BMD) as a possible…

Satellos nominates SAT-3247 as lead treatment candidate for DMD

Satellos Bioscience is hoping to bring SAT-3247, its newly nominated lead treatment candidate for Duchenne muscular dystrophy (DMD), into clinical trials next year. The company announced that it’s working to establish a good manufacturing practice (GMP) setup to make the medication, and is in the midst of experiments…

Sarepta planning to seek expanded Elevidys approval after EMBARK

While a Phase 3 clinical trial of Elevidys (delandistrogene moxeparvovec-rokl) has failed to meet its main goal, the results nonetheless showed that, compared with a placebo, the approved gene therapy led to notable improvements across numerous measures of physical function in boys with Duchenne muscular dystrophy (DMD). Top-line findings…

DYNE-251 for DMD, DYNE-101 for DM1 showing safety in early trials

DYNE-251, an experimental treatment for Duchenne muscular dystrophy (DMD), is being well tolerated by patients in an ongoing clinical trial, according to the therapy’s developer, Dyne Therapeutics. Dyne also reported positive safety data from an ongoing trial of DYNE-101, the company’s investigational therapy for myotonic dystrophy type…