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.

December 18, 2020December 18, 2020

Zolgensma Approved to Treat Young SMA Patients in Canada

News

Health Canada has approved Novartis‘ gene therapy Zolgensma (onasemnogene abeparvovec) to treat pediatric spinal muscular atrophy (SMA) patients who have three or fewer copies of the SMN2 gene, or with infantile-onset ... Read more

December 16, 2020December 16, 2020

Paramedian Injection Approach Found to Ease Spinraza Administration

News

Administering Spinraza (nusinersen) via a particular method of injection, called the paramedian approach, can shorten procedure times and reduce the occurrence of adverse events for people with spinal muscular ... Read more

November 9, 2020November 10, 2020

Spinraza’s Benefits Mild, Transient in Infant With Severe Type 0 SMA

News

An infant with type 0 spinal muscular atrophy (SMA), a severe disease form evident before birth, began treatment with Spinraza (nusinersen) at 2 weeks old and showed mild improvement, but ... Read more

November 5, 2020November 5, 2020

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

News, syndicated

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit ... Read more

October 20, 2020October 20, 2020

Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

News, syndicated

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a ... Read more

October 19, 2020October 19, 2020

Risdiplam, Oral SMA Treatment, Up for Approval in Japan

News

Chugai Pharmaceutical, part of the Roche Group, is asking that risdiplam be approved as a daily oral treatment for spinal muscular atrophy (SMA) patients in Japan. A new drug application seeking approval, ... Read more

October 16, 2020October 16, 2020

Adults With SMA Who Sought Specialty Care After Spinraza Approval May Have More Severe Disease

News

Adults with spinal muscular atrophy (SMA) who began being seen at specialty centers after the approval of Spinraza (nusinersen) may have more severe symptoms than adults who have been followed ... Read more

September 11, 2020September 11, 2020

NICE Widens Its Zolgensma Appraisal Due to European Marketing Authorization

News

England’s National Institute for Health and Care Excellence (NICE) is expanding its appraisal of Zolgensma, a gene therapy for spinal muscular atrophy (SMA), according to a press release from SMA ... Read more

August 7, 2020August 7, 2020

Phase 2 Trial of Amifampridine in SMA Type 3 Patients Fully Enrolled

News

A clinical trial testing oral amifampridine phosphate in people with spinal muscular atrophy (SMA) type 3 who are able to walk is fully enrolled, Catalyst Pharmaceuticals announced in a press release. Marketed under ... Read more

July 15, 2020July 16, 2020

Guidance on Zolgensma in Europe Notes Lack of ‘Evidence’ for Broad Use

News

Identifying those spinal muscular atrophy (SMA) patients who will benefit most from treatment with Zolgensma requires more than assessing traditional SMA types, according to a consensus statement from a panel of European ... Read more