Administering Spinraza (nusinersen) via a particular method of injection, called the paramedian approach, can shorten procedure times and reduce…
Articles by Marisa Wexler, MS
An infant with type 0 spinal muscular atrophy (SMA), a severe disease form evident before birth, began treatment with…
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like…
Chugai Pharmaceutical, part of the Roche Group, is asking that risdiplam be approved as a daily oral treatment for …
Adults with spinal muscular atrophy (SMA) who began being seen at specialty centers after the approval of Spinraza…
England’s National Institute for Health and Care Excellence (NICE) is expanding its appraisal of Zolgensma, a gene therapy for…
A clinical trial testing oral amifampridine phosphate in people with spinal muscular atrophy (SMA) type 3 who are able to walk is…
Identifying those spinal muscular atrophy (SMA) patients who will benefit most from treatment with Zolgensma requires more than assessing traditional…
Perisynaptic Schwann cells — a specialized type of cell that helps facilitate communication between neurons and muscle cells…