Posts by: Marisa Wexler MS

Marisa Wexler MS

Marisa holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.

August 27, 2020August 27, 2020

Casimersen, Exon 45 Skipping Duchenne Therapy, Under FDA Priority Review

News

An application requesting the approval of casimersen (SRP-4045) to treat Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping has been accepted and placed under priority review by the U.S. Food and Drug ... Read more

August 18, 2020August 18, 2020

Phase 3 Trial of Pamrevlumab Enrolling DMD Patients Unable to Walk

News

The LELANTOS Phase 3 clinical trial, which is evaluating FibroGen‘s pamrevlumab in people with Duchenne muscular dystrophy (DMD) who are no longer able to walk, has enrolled a first ... Read more

August 6, 2020August 6, 2020

MD Advocates Offer Advice for Students With Neuromuscular Disorders Amid COVID-19

News

The importance of communication and self-care for people with muscular dystrophy and other neuromuscular diseases who are pursuing their education during the COVID-19 pandemic was highlighted in a Facebook ... Read more

July 30, 2020August 14, 2020

FDA Places SRP-9001, DMD Micro-dystrophin Gene Therapy, on Fast Track

News

An investigational gene therapy for Duchenne muscular dystrophy (DMD), called SRP-9001 micro-dystrophin, was given fast track designation by the U.S. Food and Drug Administration (FDA) its developer, Sarepta Therapeutics, announced in ... Read more

July 9, 2020July 9, 2020

UK Renews Puldysa Early Access Program for Duchenne Patients

News

Oral Puldysa (idebenone), Santhera Pharmaceuticals‘ investigational treatment for breathing problems caused by Duchenne muscular dystrophy (DMD), will be available to eligible patients in the U.K. for another year via ... Read more

June 4, 2020June 4, 2020

Santhera and Rutgers Partner on Gene Therapy Work for LAMA2 MD

News

Santhera Pharmaceuticals announced the signing of two agreements with Rutgers University related to the development of potential gene therapies for LAMA2-deficient congenital muscular dystrophy (LAMA2 MD). Included are a license agreement as ... Read more

April 30, 2020April 30, 2020

PTC and Sarepta Say MD Treatment Supplies Uninterrupted, Encourage Use of Patient Support Programs

News

Both PTC Therapeutics and Sarepta Therapeutics expect to continue providing an uninterrupted supply of their Duchenne muscular dystrophy (DMD) therapies during the COVID-19 pandemic, representatives from the companies said in a ... Read more

April 28, 2020April 28, 2020

MDA’s Medical Advisory Team to Help Guide Research and Patient Care

News

The Muscular Dystrophy Association (MDA) has formed a Medical Advisory Team to provide guidance related to research and clinical care in muscular dystrophy, amyotrophic lateral sclerosis (ALS) and other neuromuscular ... Read more

April 23, 2020April 30, 2020

COVID-19 Delays Phase 3 Trial of Givinostat in DMD, Italfarmaco Says in Webinar

News

Because of the global COVID-19 pandemic, Italfarmaco has paused enrollment of boys with Duchenne muscular dystrophy (DMD) in a Phase 3 trial of its investigational treatment givinostat, the company ... Read more

April 2, 2020April 2, 2020

MDA Hosting Online Q&A on COVID-19 Today

News

The Muscular Dystrophy Association (MDA) is hosting an online Q&A session today to discuss the best ways that people with neuromuscular diseases might protect themselves during the COVID-19 pandemic. ... Read more