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.

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

July 1, 2020July 1, 2020

New Glia Cell Markers May Provide Insight Into Neuromuscular Diseases, Study Says

News

Perisynaptic Schwann cells — a specialized type of cell that helps facilitate communication between neurons and muscle cells — express specific molecular markers, which could make it easier to ... Read more

June 26, 2020June 26, 2020

Disparities Found in Parents, Children’s Perceptions of SMA

News

Perceptions about illness often don’t line up between children with spinal muscular atrophy (SMA) and their parents, with mothers and fathers often perceiving disease severity as worse than their ... Read more

June 12, 2020July 23, 2020

Cure SMA Details Its ‘Clinical Trial Readiness Program’

News

A readiness program to help clinical trial sites prepare to run spinal muscular atrophy (SMA) studies has been established by Cure SMA in collaboration with the pharmaceutical industry, other patient ... Read more

June 10, 2020June 10, 2020

Interviews Probe How Unaffected Siblings Learn Genetic Implications of SMA

News

Siblings of people with spinal muscular atrophy (SMA) who do not have the disease themselves tend to learn about it gradually over time from both their parents and external ... Read more

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